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1. Long-term effect of thymectomy plus prednisone versus prednisone alone in patients with non-thymomatous myasthenia gravis: 2-year extension of the MGTX randomised trial

Author

Wolfe, Gil I, Kaminski, Henry J, Aban, Inmaculada B, Minisman, Greg, Kuo, Hui-Chien, Marx, Alexander, Ströbel, Philipp, Mazia, Claudio, Oger, Joel, Cea, J Gabriel, Heckmann, Jeannine M, Evoli, Amelia, Nix, Wilfred, Ciafaloni, Emma, Antonini, Giovanni, Witoonpanich, Rawiphan, King, John O, Beydoun, Said R, Chalk, Colin H, Barboi, Alexandru C, Amato, Anthony A, Shaibani, Aziz I, Katirji, Bashar, Lecky, Bryan RF, Buckley, Camilla, Vincent, Angela, Dias-Tosta, Elza, Yoshikawa, Hiroaki, Waddington-Cruz, Márcia, Pulley, Michael T, Rivner, Michael H, Kostera-Pruszczyk, Anna, Pascuzzi, Robert M, Jackson, Carlayne E, Verschuuren, Jan JGM, Massey, Janice M, Kissel, John T, Werneck, Lineu C, Benatar, Michael, Barohn, Richard J, Tandan, Rup, Mozaffar, Tahseen, Silvestri, Nicholas J, Conwit, Robin, Sonett, Joshua R, Jaretzki, Alfred, Newsom-Davis, John, Cutter, Gary R, Group, MGTX Study, Cutter, Gary, Aban, Inmaculada, Feese, Michelle, Wolfe, Gil, Kaminski, Henry, Sonett, Joshua, Saluto, Valeria, Rosenberg, Moises, Alvarez, Valeria, Rey, Lisa, King, John, Butzkueven, Helmut, Goldblatt, John, Carey, John, Pollard, John, Reddel, Stephen, Handel, Nicholas, McCaughan, Brian, Pallot, Linda, Novis, Ricardo, Boasquevisque, Carlos, Morato-Fernandez, Rubens, Ximenes, Manoel, Werneck, Lineu, Scola, Rosana, Soltoski, Paulo, Chalk, Colin, Moore, Fraser, Mulder, David, Wadup, Lisa, Mezei, Michele, Evans, Kenneth, Jiwa, Theresa, Schaffar, Anne, White, Chris, Toth, Cory, Gelfand, Gary, Wood, Susan, Pringle, Elizabeth, Zwicker, Jocelyn, Maziak, Donna, Shamji, Farid, and Sundaresan, Sudhir

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Neurosciences, Myasthenia Gravis, Autoimmune Disease, Rare Diseases, Clinical Research, Clinical Trials and Supportive Activities, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Adult, Female, Humans, Longitudinal Studies, Male, Prednisone, Thymectomy, Treatment Outcome, Young Adult, MGTX Study Group, Neurology & Neurosurgery, Clinical sciences
Abstract

BackgroundThe Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone (MGTX) showed that thymectomy combined with prednisone was superior to prednisone alone in improving clinical status as measured by the Quantitative Myasthenia Gravis (QMG) score in patients with generalised non-thymomatous myasthenia gravis at 3 years. We investigated the long-term effects of thymectomy up to 5 years on clinical status, medication requirements, and adverse events.MethodsWe did a rater-blinded 2-year extension study at 36 centres in 15 countries for all patients who completed the randomised controlled MGTX and were willing to participate. MGTX patients were aged 18 to 65 years at enrolment, had generalised non-thymomatous myasthenia gravis of less than 5 years' duration, had acetylcholine receptor antibody titres of 1·00 nmol/L or higher (or concentrations of 0·50-0·99 nmol/L if diagnosis was confirmed by positive edrophonium or abnormal repetitive nerve stimulation, or abnormal single fibre electromyography), had Myasthenia Gravis Foundation of America Clinical Classification Class II-IV disease, and were on optimal anticholinesterase therapy with or without oral corticosteroids. In MGTX, patients were randomly assigned (1:1) to either thymectomy plus prednisone or prednisone alone. All patients in both groups received oral prednisone at doses titrated up to 100 mg on alternate days until they achieved minimal manifestation status. The primary endpoints of the extension phase were the time-weighted means of the QMG score and alternate-day prednisone dose from month 0 to month 60. Analyses were by intention to treat. The trial is registered with ClinicalTrials.gov, number NCT00294658. It is closed to new participants, with follow-up completed.FindingsOf the 111 patients who completed the 3-year MGTX, 68 (61%) entered the extension study between Sept 1, 2009, and Aug 26, 2015 (33 in the prednisone alone group and 35 in the prednisone plus thymectomy group). 50 (74%) patients completed the 60-month assessment, 24 in the prednisone alone group and 26 in the prednisone plus thymectomy group. At 5 years, patients in the thymectomy plus prednisone group had significantly lower time-weighted mean QMG scores (5·47 [SD 3·87] vs 9·34 [5·08]; p=0·0007) and mean alternate-day prednisone doses (24 mg [SD 21] vs 48 mg [29]; p=0·0002) than did those in the prednisone alone group. 14 (42%) of 33 patients in the prednisone group, and 12 (34%) of 35 in the thymectomy plus prednisone group, had at least one adverse event by month 60. No treatment-related deaths were reported during the extension phase.InterpretationAt 5 years, thymectomy plus prednisone continues to confer benefits in patients with generalised non-thymomatous myasthenia gravis compared with prednisone alone. Although caution is appropriate when generalising our findings because of the small sample size of our study, they nevertheless provide further support for the benefits of thymectomy in patients with generalised non-thymomatous myasthenia gravis.FundingNational Institutes of Health, National Institute of Neurological Disorders and Stroke.

Published
2019

2. Randomized Trial of Thymectomy in Myasthenia Gravis

Author

Wolfe, Gil I, Kaminski, Henry J, Aban, Inmaculada B, Minisman, Greg, Kuo, Hui-Chien, Marx, Alexander, Ströbel, Philipp, Mazia, Claudio, Oger, Joel, Cea, J Gabriel, Heckmann, Jeannine M, Evoli, Amelia, Nix, Wilfred, Ciafaloni, Emma, Antonini, Giovanni, Witoonpanich, Rawiphan, King, John O, Beydoun, Said R, Chalk, Colin H, Barboi, Alexandru C, Amato, Anthony A, Shaibani, Aziz I, Katirji, Bashar, Lecky, Bryan RF, Buckley, Camilla, Vincent, Angela, Dias-Tosta, Elza, Yoshikawa, Hiroaki, Waddington-Cruz, Márcia, Pulley, Michael T, Rivner, Michael H, Kostera-Pruszczyk, Anna, Pascuzzi, Robert M, Jackson, Carlayne E, Garcia Ramos, Guillermo S, Verschuuren, Jan JGM, Massey, Janice M, Kissel, John T, Werneck, Lineu C, Benatar, Michael, Barohn, Richard J, Tandan, Rup, Mozaffar, Tahseen, Conwit, Robin, Odenkirchen, Joanne, Sonett, Joshua R, Jaretzki, Alfred, Newsom-Davis, John, and Cutter, Gary R

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Myasthenia Gravis, Clinical Trials and Supportive Activities, Neurosciences, Autoimmune Disease, Clinical Research, Rare Diseases, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Adolescent, Adult, Aged, Combined Modality Therapy, Female, Glucocorticoids, Hospitalization, Humans, Male, Middle Aged, Prednisone, Severity of Illness Index, Single-Blind Method, Thymectomy, Treatment Outcome, Young Adult, MGTX Study Group, Medical and Health Sciences, General & Internal Medicine, Biomedical and clinical sciences, Health sciences
Abstract

BackgroundThymectomy has been a mainstay in the treatment of myasthenia gravis, but there is no conclusive evidence of its benefit. We conducted a multicenter, randomized trial comparing thymectomy plus prednisone with prednisone alone.MethodsWe compared extended transsternal thymectomy plus alternate-day prednisone with alternate-day prednisone alone. Patients 18 to 65 years of age who had generalized nonthymomatous myasthenia gravis with a disease duration of less than 5 years were included if they had Myasthenia Gravis Foundation of America clinical class II to IV disease (on a scale from I to V, with higher classes indicating more severe disease) and elevated circulating concentrations of acetylcholine-receptor antibody. The primary outcomes were the time-weighted average Quantitative Myasthenia Gravis score (on a scale from 0 to 39, with higher scores indicating more severe disease) over a 3-year period, as assessed by means of blinded rating, and the time-weighted average required dose of prednisone over a 3-year period.ResultsA total of 126 patients underwent randomization between 2006 and 2012 at 36 sites. Patients who underwent thymectomy had a lower time-weighted average Quantitative Myasthenia Gravis score over a 3-year period than those who received prednisone alone (6.15 vs. 8.99, P

Published
2016

3. Choline Acetyltransferase Mutations Causing Congenital Myasthenic Syndrome: Molecular Findings and Genotype–Phenotype Correlations

Author

Arredondo, Juan, Lara, Marian, Gospe, Sídney M, Mazia, Claudio G, Vaccarezza, Maria, Garcia-Erro, Marcela, Bowe, Constance M, Chang, Celia H, Mezei, Michelle M, and Maselli, Ricardo A

Subjects
Biochemistry and Cell Biology, Biological Sciences, Rare Diseases, Genetics, Myasthenia Gravis, Neurosciences, Aetiology, 2.1 Biological and endogenous factors, Adolescent, Alleles, Amino Acid Substitution, Binding Sites, Catalytic Domain, Child, Preschool, Choline O-Acetyltransferase, DNA Mutational Analysis, Enzyme Activation, Female, Gene Expression, Genetic Association Studies, Genotype, HEK293 Cells, Humans, Hydrogen Bonding, Male, Models, Molecular, Mutation, Myasthenic Syndromes, Congenital, Phosphorylation, Protein Conformation, Substrate Specificity, ChAT, phosphorylation, enzyme kinetics, genotype-phenotype correlations, Clinical Sciences, Genetics & Heredity, Clinical sciences
Abstract

Choline acetyltransferase catalyzes the synthesis of acetylcholine at cholinergic nerves. Mutations in human CHAT cause a congenital myasthenic syndrome due to impaired synthesis of ACh; this severe variant of the disease is frequently associated with unexpected episodes of potentially fatal apnea. The severity of this condition varies remarkably, and the molecular factors determining this variability are poorly understood. Furthermore, genotype-phenotype correlations have been difficult to establish in patients with biallelic mutations. We analyzed the protein expression of phosphorylated ChAT of seven CHAT mutations, p.Val136Met, p.Arg207His, p.Arg186Trp, p.Val194Leu, p.Pro211Ala, p.Arg566Cys, and p.Ser694Cys, in HEK-293 cells to phosphorylated ChAT, determined their enzyme kinetics and thermal stability, and examined their structural changes. Three mutations, p.Arg207His, p.Arg186Trp, and p.Arg566Cys, are novel, and p.Val136Met and p.Arg207His are homozygous in three families and associated with severe disease. The characterization of mutants showed a decrease in the overall catalytic efficiency of ChAT; in particular, those located near the active-site tunnel produced the most seriously disruptive phenotypic effects. On the other hand, p.Val136Met, which is located far from both active and substrate-binding sites, produced the most drastic reduction of ChAT expression. Overall, CHAT mutations producing low enzyme expression and severe kinetic effects are associated with the most severe phenotypes.

Published
2015

4. Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study

Author

Mazia, Claudio Gabriel, Wilken, Miguel, Barroso, Fabio, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Chaves, Marcelo, Vidal, Gonzalo, Garcia, Alejandra Dalila, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, Mercelis, Rudy, Mahieu, Délphine, Wagemaekers, Linda, Van Damme, Philip, Depreitere, Annelies, Schotte, Caroline, Smetcoren, Charlotte, Stevens, Olivier, Van Daele, Sien, Vandenbussche, Nicolas, Vanhee, Annelies, Verjans, Sarah, Vynckier, Jan, D'Hondt, Ann, Tilkin, Petra, Alves de Siqueira Carvalho, Alzira, Dias Brockhausen, Igor, Feder, David, Ambrosio, Daniel, César, Pamela, Melo, Ana Paula, Martins Ribeiro, Renata, Rocha, Rosana, Bezerra Rosa, Bruno, Veiga, Thabata, da Silva, Luiz Augusto, Santos Engel, Murilo, Gonçalves Geraldo, Jordana, Ananias Morita, Maria da Penha, Nogueira Coelho, Erica, Paiva, Gabriel, Pozo, Marina, Prando, Natalia, Martineli Torres, Debora Dada, Butinhao, Cristiani Fernanda, Duran, Gustavo, Gomes da Silva, Tamires Cristina, Otavio Maia Gonçalves, Luiz, Pazetto, Lucas Eduardo, Fialho, Tomás Augusto Suriane, Renata Cubas Volpe, Luciana, Souza Duca, Luciana, Souza Bulle Oliveira, Acary, Amaral Andrade, Ana Carolina, Annes, Marcelo, Duarte Silva, Liene, Cavalcante Lino, Valeria, Pinto, Wladimir, Assis, Natália, Carrara, Fernanda, Miranda, Carolina, Souza, Iandra, Fernandes, Patricia, Siddiqi, Zaeem, Phan, Cecile, Narayan, Jeffrey, Blackmore, Derrick, Mallon, Ashley, Roderus, Rikki, Watt, Elizabeth, Junkerova, Jana, Kurkova, Barbora, Reguliova, Katarina, Zapletalova, Olga, Pitha, Jiri, Novakova, Iveta, Tyblova, Michaela, Jurajdova, Ivana, Wolfova, Marcela, Andersen, Henning, Harbo, Thomas, Vinge, Lotte, Krogh, Susanne, Mogensen, Anita, Vissing, John, Højgaard, Joan, Witting, Nanna, Mette Ostergaard Autzen, Anne, Pedersen, Jane, Eralinna, Juha-Pekka, Laaksonen, Mikko, Oksaranta, Olli, Harrison, Tuula, Eriksson, Jaana, Rozsa, Csilla, Horvath, Melinda, Lovas, Gabor, Matolcsi, Judit, Szabo, Gyorgyi, Jakab, Gedeonne, Szabadosne, Brigitta, Antonini, Giovanni, Di Pasquale, Antonella, Garibaldi, Matteo, Morino, Stefania, Troili, Fernanda, Fionda, Laura, Filla, Allessandro, Costabile, Teresa, Marano, Enrico, Saccà, Francesco, Fasanaro, Angiola, Marsili, Angela, Puorro, Giorgia, Mantegazza, Renato, Antozzi, Carlo, Bonanno, Silvia, Camera, Giorgia, Locatelli, Alberta, Maggi, Lorenzo, Pasanisi, Maria, Campanella, Angela, Evoli, Amelia, Alboini, Paolo Emilio, D'Amato, Valentina, Iorio, Raffaele, Kanai, Tetsuya, Kawaguchi, Naoki, Mori, Masahiro, Kaneko, Yoko, Kanzaki, Akiko, Kobayashi, Eri, Masaki, Katsuhisa, Matsuse, Dai, Matsushita, Takuya, Uehara, Taira, Shimpo, Misa, Jingu, Maki, Kikutake, Keiko, Nakamura, Yumiko, Sano, Yoshiko, Nagane, Yuriko, Kamegamori, Ikuko, Tsuda, Tomoko, Fujii, Yuko, Futono, Kazumi, Ozawa, Yukiko, Mizugami, Aya, Saito, Yuka, Morikawa, Miyuki, Samukawa, Makoto, Kamakura, Sachiko, Miyawaki, Eriko, Mitazaki, Teiichiro, Motomura, Masakatsu, Mukaino, Akihiro, Yoshimura, Shunsuke, Asada, Shizuka, Yoshida, Seiko, Amamoto, Shoko, Kobashikawa, Tomomi, Koga, Megumi, Maeda, Yasuko, Takada, Kazumi, Takada, Mihoko, Tsurumaru, Masako, Yamashita, Yumi, Akiyama, Tetsuya, Narikawa, Koichi, Tano, Ohito, Tsukita, Kenichi, Kurihara, Rikako, Meguro, Fumie, Fukuda, Yusuke, Sato, Miwako, Funaka, Soichiro, Kawamura, Tomohiro, Makamori, Masayuki, Takahashi, Masanori, Taichi, Namie, Hasuike, Tomoya, Higuchi, Eriko, Kobayashi, Hisako, Osakada, Kaori, Tsuda, Emiko, Shimohama, Shun, Hayashi, Takashi, Hisahara, Shin, Kawamata, Jun, Murahara, Takashi, Saitoh, Masaki, Suzuki, Shuichiro, Yamamoto, Daisuke, Ishiyama, Yoko, Ishiyama, Naoko, Noshiro, Mayuko, Takeyama, Rumi, Uwasa, Kaori, Yasuda, Ikuko, van der Kooi, Anneke, de Visser, Marianne, Gibson, Tamar, Casasnovas, Carlos, Alberti Aguilo, Maria Antonia, Homedes-Pedret, Christian, Julia Palacios, Natalia, Diez Porras, Laura, Velez Santamaria, Valentina, Lazaro, Ana, Diez Tejedor, Exuperio, Gomez Salcedo, Pilar, Fernandez-Fournier, Mireya, Lopez Ruiz, Pedro, Rodriguez de Rivera, Francisco Javier, Sastre, Maria, Gamez, Josep, Sune, Pilar, Salvado, Maria, Gili, Gisela, Mazuela, Gonzalo, Illa, Isabel, Cortes Vicente, Elena, Diaz-Manera, Jordi, Querol Gutierrez, Luis Antonio, Rojas Garcia, Ricardo, Vidal, Nuria, Arribas-Ibar, Elisabet, Piehl, Fredrik, Hietala, Albert, Bjarbo, Lena, Sengun, Ihsan, Meherremova, Arzu, Ozcelik, Pinar, Balkan, Bengu, Tuga, Celal, Ugur, Muzeyyen, Erdem-Ozdamar, Sevim, Bekircan-Kurt, Can Ebru, Acar, Nazire Pinar, Yilmaz, Ezgi, Caliskan, Yagmur, Orsel, Gulsah, Efendi, Husnu, Aydinlik, Seda, Cavus, Hakan, Kutlu, Ayse, Becerikli, Gulsah, Semiz, Cansu, Tun, Ozlem, Terzi, Murat, Dogan, Baki, Onar, Musa Kazim, Sen, Sedat, Kirbas Cavdar, Tugce, Veske, Adife, Norwood, Fiona, Dimitriou, Aikaterini, Gollogly, Jakit, Mahdi-Rogers, Mohamed, Seddigh, Arshira, Sokratous, Giannis, Maier, Gal, Sohail, Faisal, Jacob, Saiju, Sadalage, Girija, Torane, Pravin, Brown, Claire, Shah, Amna, Sathasivam, Sivakumar, Arndt, Heike, Davies, Debbie, Watling, Dave, Amato, Anthony, Cochrane, Thomas, Salajegheh, Mohammed, Roe, Kristen, Amato, Katherine, Toska, Shirli, Wolfe, Gil, Silvestri, Nicholas, Patrick, Kara, Zakalik, Karen, Katz, Jonathan, Miller, Robert, Engel, Marguerite, Forshew, Dallas, Bravver, Elena, Brooks, Benjamin, Plevka, Sarah, Burdette, Maryanne, Cunningham, Scott, Sanjak, Mohammad, Kramer, Megan, Nemeth, Joanne, Schommer, Clara, Tierney, Scott, Juel, Vern, Guptill, Jeffrey, Hobson-Webb, Lisa, Massey, Janice, Beck, Kate, Carnes, Donna, Loor, John, Anderson, Amanda, Pascuzzi, Robert, Bodkin, Cynthia, Kincaid, John, Snook, Riley, Guingrich, Sandra, Micheels, Angela, Chaudhry, Vinay, Corse, Andrea, Mosmiller, Betsy, Kelley, Andrea, Ho, Doreen, Srinivasan, Jayashri, Vytopil, Michal, Jara, Jordan, Ventura, Nicholas, Scala, Stephanie, Carter, Cynthia, Donahue, Craig, Herbert, Carol, Weiner, Elaine, Alam, Sharmeen, McKinnon, Jonathan, Haar, Laura, McKinnon, Naya, Alcon, Karan, McKenna, Kaitlyn, Sattar, Nadia, Daniels, Kevin, Jeffery, Dennis, Freimer, Miriam, Hoyle, Joseph Chad, Kissel, John, Agriesti, Julie, Chelnick, Sharon, Mezache, Louisa, Pineda, Colleen, Muharrem, Filiz, Karam, Chafic, Khoury, Julie, Marburger, Tessa, Kaur, Harpreet, Dimitrova, Diana, Gilchrist, James, Agrawal, Brajesh, Elsayed, Mona, Kohlrus, Stephanie, Andoin, Angela, Darnell, Taylor, Golden, Laura, Lokaitis, Barbara, Seelback, Jenna, Muppidi, Srikanth, Goyal, Neelam, Sakamuri, Sarada, So, Yuen T., Paulose, Shirley, Pol, Sabrina, Welsh, Lesly, Bhavaraju-Sanka, Ratna, Tobon Gonzales, Alejandro, Dishman, Lorraine, Jones, Floyd, Gonzalez, Anna, Padilla, Patricia, Saklad, Amy, Silva, Marcela, Kazamel, Mohamed, Alsharabati, Mohammad, Lu, Liang, Nozaki, Kenkichi, Mumfrey-Thomas, Sandi, Woodall, Amy, Mozaffar, Tahseen, Cash, Tiyonnoh, Goyal, Namita, Roy, Gulmohor, Mathew, Veena, Maqsood, Fatima, Minton, Brian, Jones, H. James, Rosenfeld, Jeffrey, Garcia, Rebekah, Echevarria, Laura, Garcia, Sonia, Pulley, Michael, Aranke, Shachie, Berger, Alan Ross, Shah, Jaimin, Shabbir, Yasmeen, Smith, Lisa, Varghese, Mary, Gutmann, Laurie, Gutmann, Ludwig, Jerath, Nivedita, Nance, Christopher, Swenson, Andrea, Olalde, Heena, Kressin, Nicole, Sieren, Jeri, Barohn, Richard, Dimachkie, Mazen, Glenn, Melanie, McVey, April, Pasnoor, Mamatha, Statland, Jeffery, Wang, Yunxia, Liu, Tina, Emmons, Kelley, Jenci, Nicole, Locheke, Jerry, Fondaw, Alex, Johns, Kathryn, Rico, Gabrielle, Walsh, Maureen, Herbelin, Laura, Hafer-Macko, Charlene, Kwan, Justin, Zilliox, Lindsay, Callison, Karen, Young, Valerie, DiSanzo, Beth, Naunton, Kerry, Benatar, Michael, Bilsker, Martin, Sharma, Khema, Cooley, Anne, Reyes, Eliana, Michon, Sara-Claude, Sheldon, Danielle, Steele, Julie, Howard Jr, James, Chopra, Manisha, Traub, Rebecca, Vu, Tuan, Katzin, Lara, McClain, Terry, Harvey, Brittany, Hart, Adam, Huynh, Kristin, Beydoun, Said, Chilingaryan, Amaiak, Doan, Victor, Droker, Brian, Gong, Hui, Karimi, Sanaz, Lin, Frank, Pokala, Krishna, Shah, Akshay, Tran, Anh, Akhter, Salma, Malekniazi, Ali, Tandan, Rup, Hehir, Michael, Waheed, Waqar, Lucy, Shannon, Weiss, Michael, Distad, Jane, Strom, Susan, Downing, Sharon, Kim, Bryan, Nowak, Richard, Dicapua, Daniel, Keung, Benison, Kumar, Aditya, Patwa, Huned, Robeson, Kimberly, Yang, Irene, Nye, Joan, Vu, Hong, Murai, Hiroyuki, Uzawa, Akiyuki, Suzuki, Yasushi, Imai, Tomihiro, Shiraishi, Hirokazu, Suzuki, Hidekazu, Okumura, Meinoshin, O’Brien, Fanny, Wang, Jing-Jing, Fujita, Kenji P., and Utsugisawa, Kimiaki

Published
2019
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5. Long-term effect of thymectomy plus prednisone versus prednisone alone in patients with non-thymomatous myasthenia gravis: 2-year extension of the MGTX randomised trial

Author

Cutter, Gary, Aban, Inmaculada, Minisman, Greg, Feese, Michelle, Kuo, Hui-Chien, Newsom-Davis, John, Wolfe, Gil, Kaminski, Henry, Jaretzki, Alfred, Sonett, Joshua, Mazia, Claudio, Saluto, Valeria, Rosenberg, Moises, Alvarez, Valeria, Rey, Lisa, King, John, Butzkueven, Helmut, Goldblatt, John, Carey, John, Pollard, John, Reddel, Stephen, Handel, Nicholas, McCaughan, Brian, Pallot, Linda, Waddington-Cruz, Márcia, Novis, Ricardo, Boasquevisque, Carlos, Dias-Tosta, Elza, Morato-Fernandez, Rubens, Ximenes, Manoel, Werneck, Lineu, Scola, Rosana, Soltoski, Paulo, Chalk, Colin, Moore, Fraser, Mulder, David, Wadup, Lisa, Oger, Joel, Mezei, Michele, Evans, Kenneth, Jiwa, Theresa, Schaffar, Anne, White, Chris, Toth, Cory, Gelfand, Gary, Wood, Susan, Pringle, Elizabeth, Zwicker, Jocelyn, Maziak, Donna, Shamji, Farid, Sundaresan, Sudhir, Seely, Andrew, Cea, Gabriel, Verduga, Renato, Aguayo, Alberto, Jander, Sebastian, Zickler, Philipp, Klein, Michael, Marx, Alexander, Ströbel, Philipp, Weis, Cleo-Aron, Melms, Arthur, Bischof, Felix, Aebert, Hermann, Ziemer, Gerhard, Nix, Wilfred, Thümler, Björn, Wilhem-Schwenkmezger, Thomas, Mayer, Eckhard, Schalke, Berthold, Pöschel, Peter, Hieber, Gisela, Wiebe, Karsten, Antonini, Giovanni, Clemenzi, Alessandro, Ceschin, Vanessa, Rendina, Erino, Venuta, Federico, Morino, Stefania, Bucci, Elisabetta, Durelli, Luca, Tavella, Alessia, Clerico, Marinella, Contessa, Giulia, Borasio, Piero, Evoli, Amelia, Servidei, Serenella, Granone, Pierluigi, Mantegazza, Renato, Berta, Emilia, Novellino, Lorenzo, Spinelli, Luisa, Motomura, Masakatsu, Matsuo, Hidenori, Nagayasu, Takeshi, Yoshikawa, Hiroaki, Takamori, Masaharu, Oda, Makoto, Matsumoto, Isao, Furukawa, Yutaka, Noto, Daisuke, Motozaki, Yuko, Iwasa, Kazuo, Yanase, Daisuke, Garcia Ramos, Guillermo, Cacho, Bernardo, de la Garza, Lorenzo, Kostera-Pruszczyk, Anne, Lipowska, Marta, Kwiecinski, Hubert, Potulska-Chromik, Anna, Orlowski, Tadeusz, Silva, Ana, Feijo, Marta, Freitas, António, Heckmann, Jeannine, Frost, Andrew, Pan, Edward, Tucker, Lawrence, Rossouw, Johan, Drummond, Fiona, Illa, Isabel, Diaz, Jorge, Leon, Carlos, Yeh, Jiann-Horng, Chiu, Hou-Chang, Hsieh, Yei-San, Witoonpanich, Rawiphan, Tunlayadechanont, Supoch, Attanavanich, Sukasom, Verschuuren, Jan, Straathof, Chiara, Titulaer, Maarten, Versteegh, Michel, Pels, Arda, Krum, Yvonne, Buckley, Camilla, Leite, M. Isabel, Vincent, Angela, Hilton-Jones, David, Ratnatunga, Chandi, Farrugia, Maria, Petty, Richard, Overell, James, Kirk, Alan, Gibson, Andrew, McDermott, Chris, Hopkinson, David, Lecky, Bryan, Watling, David, Marshall, Dot, Saminaden, Sam, Davies, Deborah, Dougan, Charlotte, Sathasivam, Siva, Page, Richard, Sussman, Jon, Ealing, John, Krysiak, Peter, Amato, Anthony, Salajegheh, Mohammad, Jaklitsch, Michael, Roe, Kristen, Ashizawa, Tetsuo, Smith, Robert Glenn, Zwischenberg, Joseph, Stanton, Penny, Barboi, Alexandru, Jaradeh, Safwan, Tisol, William, Gasparri, Mario, Haasler, George, Yellick, Mary, Dennis, Cedric, Barohn, Richard, Pasnoor, Mamatha, Dimachkie, Mazen, McVey, April, Gronseth, Gary, Dick, Arthur, Kramer, Jeffrey, Currence, Melissa, Herbelin, Laura, Belsh, Jerry, Li, George, Langenfeld, John, Mertz, Mary Ann, Benatar, Michael, Harrison, Taylor, Force, Seth, Usher, Sharon, Beydoun, Said, Lin, Frank, DeMeester, Steve, Akhter, Salem, Malekniazi, Ali, Avenido, Gina, Crum, Brian, Milone, Margherita, Cassivi, Stephen, Fisher, Janet, Ciafaloni, Emma, Heatwole, Chad, Watson, Thomas, Hilbert, James, Smirnow, Alexis, Distad, B. Jane, Weiss, Michael, Wood, Douglas, Haug, Joanna, Ernstoff, Raina, Cao, Jingyang, Chmielewski, Gary, Welsh, Robert, Duris, Robin, Gutmann, Laurie, Pawar, Gauri, Graeber, Geoffrey Marc, Altemus, Patricia, Nance, Christopher, Gutmann, Ludwig, Jackson, Carlayne, Grogan, Patrick, Calhoon, John, Kittrell, Pamela, Myers, Deborah, Hayat, Ghazala, Naunheim, Keith, Eller, Susan, Holzemer, Eve, Katirji, Bashar, Alshekhlee, Amer, Robke, Jason, Karlinchak, Brenda, Katz, Jonathan, Miller, Robert, Roan, Ralph, Forshew, Dallas, Kissel, John, Elsheikh, Bakri, Ross, Patrick, Chelnick, Sharon, Lewis, Richard, Acsadi, Agnes, Baciewicz, Frank, Masse, Stacey, Massey, Janice, Juel, Vern, Onaitis, Mark, Lowe, James, Lipscomb, Bernadette, Mozaffar, Tahseen, Thai, Gaby, Milliken, Jeffrey, Martin, Veronica, Karayan, Ronnie, Muley, Suraj, Parry, Gareth, Shumway, Sara, Oh, Shin, Claussen, Gwen, Lu, Liang, Cerfolio, Robert, Young, Angela, Morgan, Marla, Pascuzzi, Robert, Kincaid, John, Kesler, Kenneth, Guingrich, Sandy, Michaels, Angi, Phillips, Lawrence, Burns, Ted, Jones, David, Fischer, Cindy, Pulley, Michael, Berger, Alan, D'Agostino, Harry, Smith, Lisa, Rivner, Michael, Pruitt, Jerry, Landolfo, Kevin, Hillman, Demetric, Shaibani, Aziz, Sermas, Angelo, Ruel, Ross, Ismail, Farah, Sivak, Mark, Goldstein, Martin, Camunas, Jorge, Bratton, Joan, Tandan, Rup, Panitch, Hill, Leavitt, Bruce, Jones, Marilee, Muppidi, Srikanth, Vernino, Steven, Nations, Sharon, Meyer, Dan, Gorham, Nina, Wolfe, Gil I, Kaminski, Henry J, Aban, Inmaculada B, Cea, J Gabriel, Heckmann, Jeannine M, King, John O, Beydoun, Said R, Chalk, Colin H, Barboi, Alexandru C, Amato, Anthony A, Shaibani, Aziz I, Lecky, Bryan R F, Pulley, Michael T, Rivner, Michael H, Kostera-Pruszczyk, Anna, Pascuzzi, Robert M, Jackson, Carlayne E, Verschuuren, Jan J G M, Massey, Janice M, Kissel, John T, Werneck, Lineu C, Barohn, Richard J, Silvestri, Nicholas J, Conwit, Robin, Sonett, Joshua R, Jaretzki, Alfred, III, and Cutter, Gary R

Published
2019
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6. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study

Author

Mazia, Claudio Gabriel, Wilken, Miguel, Ortea, Carolina, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Vidal, Gonzalo, Garcia, Alejandra Dalila, Lamont, Phillipa, Leong, Wai-Kuen, Boterhoven, Heidi, Fyfe, Beverly, Roberts, Leslie, Jasinarachchi, Mahi, Willlems, Natasha, Wanschitz, Julia, Löscher, Wolfgang, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, Mercelis, Rudy, Wagemaekers, Linda, Mahieu, Delphine, Van Damme, Philip, Smetcoren, Charlotte, Stevens, Olivier, Verjans, Sarah, D'Hondt, Ann, Tilkin, Petra, Alves de Siqueira Carvalho, Alzira, Hasan, Rosa, Dias Brockhausen, Igor, Feder, David, Ambrosio, Daniel, Melo, Ana Paula, Rocha, Rosana, Rosa, Bruno, Veiga, Thabata, Augusto da Silva, Luiz, Gonçalves Geraldo, Jordana, da Penha Morita Ananias, Maria, Nogueira Coelho, Erica, Paiva, Gabriel, Pozo, Marina, Prando, Natalia, Dada Martineli Torres, Debora, Fernanda Butinhao, Cristiani, Coelho, Erica, Renata Cubas Volpe, Luciana, Duran, Gustavo, Gomes da Silva, Tamires Cristina, Otavio Maia Gonçalves, Luiz, Pazetto, Lucas Eduardo, Souza Duca, Luciana, Suriane Fialho, Tomás Augusto, Gheller Friedrich, Maurício André, Guerreiro, Alexandre, Mohr, Henrique, Pereira Martins, Maurer, da Cruz Pacheco, Daiane, Macagnan, Ana Paula, de Cassia Santos, Aline, Bulle Oliveira, Acary Souza, Amaral de Andrade, Ana Carolina, Annes, Marcelo, Cavalcante Lino, Valeria, Pinto, Wladimir, Miranda, Carolina, Carrara, Fernanda, Souza, Iandra, Genge, Angela, Massie, Rami, Campbell, Natasha, Bril, Vera, Katzberg, Hans, Soltani, Mehran, Ng, Eduardo, Siddiqi, Zaeem, Phan, Celile, Blackmore, Derrick, Vohanka, Stanislav, Bednarik, Josef, Chmelikova, Magda, Cierny, Marek, Toncrova, Stanislava, Junkerova, Jana, Kurkova, Barbora, Reguliova, Katarina, Zapletalova, Olga, Pitha, Jiri, Novakova, Iveta, Tyblova, Michaela, Wolfova, Marcela, Jurajdova, Ivana, Andersen, Henning, Harbo, Thomas, Vinge, Lotte, Mogensen, Anita, Højgaard, Joan, Witting, Nanna, Autzen, Anne Mette, Pedersen, Jane, Färkkilä, Markus, Atula, Sari, Nyrhinen, Anne, Erälinna, Juha-Pekka, Laaksonen, Mikko, Oksaranta, Olli, Eriksson, Jaana, Harrison, Tuula, Desnuelle, Claude, Sacconi, Sabrina, Soriani, Marie-Hélène, Decressac, Sonia, Moutarde, Julie, Lahaut, Pauline, Solé, Guilhem, Le Masson, Gwendal, Wielanek-Bachelet, Anne-Cécile, Gaboreau, Morgane, Moreau, Caroline, Wilson, Amy, Vial, Christophe, Bouhour, Françoise, Gervais-Bernard, Helene, Merle, Hélène, Hourquin, Caroline, Lacour, Arnaud, Outteryck, Olivier, Vermersch, Patrick, Zephir, Hélène, Millois, Edouard, Deneve, Michel, Deruelle, Fabienne, Schoser, Benedikt, Wenninger, Stephan, Stangel, Martin, Alvermann, Sascha, Gingele, Stefan, Skripuletz, Thomas, Suehs, Kurt-Wolfram, Trebst, Corinna, Fricke, Karin, Papagiannopoulos, Sotirios, Bostantzopoulou, Sevasti, Vlaikidis, Nicholas, Zampaki, Martha, Papadopoulou, Nikoletta, Mitsikostas, Dimos-Dimitrios, Kasioti, Eleni, Mitropoulou, Efstathia, Charalambous, Despoina, Rozsa, Csilla, Horvath, Melinda, Lovas, Gabor, Matolcsi, Judit, Szabo, Gyorgyi, Szabadosne, Brigitta, Vecsei, Laszlo, Dezsi, Livia, Varga, Edina, Konyane, Monika, Gross, Bella, Azrilin, Olga, Greenbereg, Nelly, Bali Kuperman, Hila, Antonini, Giovanni, Garibaldi, Matteo, Morino, Stefania, Troili, Fernanda, Di Pasquale, Antonella, Filla, Alessandro, Costabile, Teresa, Marano, Enrico, Sacca, Francesco, Marsil, Angela, Puorro, Giorgia, Maestri Tassoni, Michelangelo, De Rosa, Anna, Bonanno, Silvia, Antozzi, Carlo, Maggi, Lorenzo, Campanella, Angela, Angelini, Corrado, Cudia, Paola, Pegoraro, Valentina, Pinzan, Elena, Bevilacqua, Francesca, Orrico, Daniele, Bonifati, Domenico Marco, Evoli, Amelia, Alboini, Paolo Emilio, D'Amato, Valentina, Iorio, Raffaele, Inghilleri, Maurizio, Fionda, Laura, Frasca, Vittorio, Giacomelli, Elena, Gori, Maria, Lopergolo, Diego, Onesti, Emanuela, Gabriele, Maria, Patti, Francesco, Salvatore Caramma, Andrea, Messina, Silvia, Reggio, Ester, Caserta, Cinzia, Uzawa, Akiyuki, Kanai, Tetsuya, Mori, Masahiro, Kaneko, Yoko, Kanzaki, Akiko, Kobayashi, Eri, Masaki, Katsuhisa, Matsuse, Dai, Matsushita, Takuya, Uehara, Taira, Shimpo, Misa, Jingu, Maki, Kikutake, Keiko, Nakamura, Yumiko, Sano, Yoshiko, Nagane, Yuriko, Kamegamori, Ikuko, Fujii, Yuko, Futono, Kazumi, Tsuda, Tomoko, Saito, Yuka, Suzuki, Hidekazu, Morikawa, Miyuki, Samukawa, Makoto, Kamakura, Sachiko, Shiraishi, Hirokazu, Mitazaki, Teiichiro, Motomura, Masakatsu, Mukaino, Akihiro, Yoshimura, Shunsuke, Asada, Shizuka, Kobashikawa, Tomomi, Koga, Megumi, Maeda, Yasuko, Takada, Kazumi, Takada, Mihoko Takada, Yamashita, Yumi, Yoshida, Seiko, Suzuki, Yasushi, Akiyama, Tetsuya, Narikawa, Koichi, Tsukita, Kenichi, Meguro, Fumie, Fukuda, Yusuke, Sato, Miwako, Matsuo, Hidenori, Fukudome, Takayasu, Gondo, Yuichiro, Maeda, Yasuhiro, Nagaishi, Akiko, Nakane, Shunya, Okubo, Yoshinori, Okumura, Meinoshin, Funaka, Soichiro, Kawamura, Tomohiro, Makamori, Masayuki, Takahashi, Masanori, Hasuike, Tomoya, Higuchi, Eriko, Kobayashi, Hisako, Osakada, Kaori, Taichi, Namie, Tsuda, Emiko, Hayashi, Takashi, Hisahara, Shin, Imai, Tomihiro, Kawamata, Jun, Murahara, Takashi, Saitoh, Masaki, Shimohama, Shun, Suzuki, Shuichiro, Yamamoto, Daisuke, Konno, Shingo, Imamura, Tomomi, Inoue, Masashi, Murata, Mayumi, Nakazora, Hiroshi, Nakayama, Ritsu, Ikeda, Yasuko, Ogawa, Miki, Shirane, Maoko, Kanda, Takashi, Kawai, Motoharu, Koga, Michiaki, Ogasawara, Junichi, Omoto, Masatoshi, Sano, Yasuteru, Arima, Hideki, Fukui, Sachie, Shimose, Shigemi, Shinozaki, Hirokazu, Watanabe, Masanori, Yoshikawa, Chieko, van der Kooi, Anneke, de Visser, Marianne, Gibson, Tamar, Maessen, Jos, de Baets, Marc, Faber, Catherine, Keijzers, Maria Johanna, Miesen, Monique, Kostera-Pruszczyk, Anna, Kaminska, Anna, Kim, Byung-Jo, Lee, Chang Nyoung, Koo, Yong Seo, Seok, Hung Youl, Kang, Hoo Nam, Ra, HyeJin, Kim, Byoung Joon, Cho, Eun Bin, Lee, HyeLim, Min, Ju-Hong, Seok, Jinmyoung, Koh, Da Yoon, Kwon, JuYoung, Lee, JiEun, Park, SangAe, Hong, Yoon-Ho, Lim, Jae-Sung, Kim, MiRi, Kim, Seung Min, Kim, Yool-hee, Lee, Hyung Seok, Shin, Ha Young, Hwang, Eun Bi, Shin, MiJu, Sazonov, Denis, Yarmoschuk, Asya, Babenko, Larisa, Malkova, Nadezhda, Melnikova, Anna, Korobko, Denis, Kosykh, Evgeniya, Pokhabov, Dmitry, Nesterova, Yulia, Abramov, Vladislav, Balyazin, Victor, Casasnovas Pons, Carlos, Alberti Aguilo, Maria, Homedes-Pedret, Christian, Palacios, Natalia Julia, Lazaro, Ana, Diez Tejedor, Exuperio, Fernandez-Fournier, Mireya, Lopez Ruiz, Pedro, Rodriguez de Rivera, Francisco Javier, Salvado Figueras, Maria, Gamez, Josep, Salvado, Maria, Cortes Vicente, Elena, Diaz-Manera, Jordi, Querol Gutierrez, Luis, Rojas Garcia, Ricardo, Vidal, Nuria, Arribas-Ibar, Elisabet, Piehl, Fredrik, Hietala, Albert, Bjarbo, Lena, Lindberg, Christopher, Jons, Daniel, Andersson, Blanka, Sengun, Ihsan, Ozcelik, Pinar, Tuga, Celal, Ugur, Muzeyyen, Boz, Cavit, Altiparmak, Didem, Gazioglu, Sibel, Ozen Aydin, Cigdem, Erdem-Ozdamar, Sevim, Bekircan-Kurt, Can Ebru, Yilmaz, Ezgi, Acar, Nazire Pinar, Caliskan, Yagmur, Efendi, Husnu, Aydinlik, Seda, Cavus, Hakan, Semiz, Cansu, Tun, Ozlem, Terzi, Murat, Dogan, Baki, Onar, Musa Kazim, Sen, Sedat, Cavdar, Tugce Kirbas, Norwood, Fiona, Dimitriou, Aikaterini, Gollogly, Jakit, Mahdi-Rogers, Mohamed, Seddigh, Arshira, Maier, Gal, Sohail, Faisal, Sathasivam, Sivakumar, Arndt, Heike, Davies, Debbie, Watling, Dave, Rivner, Michael, Hartmann, J. Edward, Quarles, Brandy, Smalley, Nicole, Amato, Anthony, Cochrane, Thomas, Salajegheh, Mohammed, Roe, Kristen, Amato, Katherine, Toska, Shirli, Wolfe, Gil, Silvestri, Nicholas, Patrick, Kara, Zakalik, Karen, Katz, Jonathan, Miller, Robert, Engel, Marguerite, Bravver, Elena, Brooks, Benjamin, Plevka, Sarah, Burdette, Maryanne, Sanjak, Mohammad, Kramer, Megan, Nemeth, Joanne, Schommer, Clara, Juel, Vern, Guptill, Jeffrey, Hobson-Webb, Lisa, Beck, Kate, Carnes, Donna, Loor, John, Anderson, Amanda, Lange, Dale, Agopian, Eliz, Goldstein, Jonathan, Manning, Erin, Kaplan, Lindsay, Holzberg, Shara, Kassebaum, Nicole, Pascuzzi, Robert, Bodkin, Cynthia, Kincaid, John, Snook, Riley, Guinrich, Sandra, Micheels, Angela, Chaudhry, Vinay, Corse, Andrea, Mosmiller, Betsy, Ho, Doreen, Srinivasan, Jayashri, Vytopil, Michael, Ventura, Nicholas, Scala, Stephanie, Carter, Cynthia, Donahue, Craig, Herbert, Carol, Weiner, Elaine, McKinnon, Jonathan, Haar, Laura, McKinnon, Naya, Alcon, Karan, Daniels, Kevin, Sattar, Nadia, Jeffery, Dennis, McKenna, Kaitlyn, Guidon, Amanda, David, William, Dheel, Christina, Levine-Weinberg, Mark, Nigro, Catherine, Simpson, Ericka, Appel, Stanley H, Lai, Eugene, Lay, Jr, Luis, Pleitez, Milvia, Halton, Sharon, Faigle, Casey, Thompson, Lisa, Sivak, Mark, Shin, Susan, Bratton, Joan, Jacobs, Daniel, Brown, Gavin, Bandukwala, Ibrez, Brown, Morris, Kane, Jennifer, Blount, Ira, Freimer, Miriam, Hoyle, J. Chad, Agriesti, Julie, Khoury, Julie, Marburger, Tessa, Kaur, Harpreet, Dimitrova, Diana, Mellion, Michelle, Sachs, George, Crabtree, Brigid, Keo, Roseann, Perez, Ele Kim, Taber, Sandra, Gilchrist, James, Andoin, Angela, Darnell, Taylor, Goyal, Neelam, Sakamuri, Sarada, So, Yuen T, Welsh, Lesly Welsh, Bhavaraju-Sanka, Ratna, Tobon Gonzalez, Alejandro, Jones, Floyd, Saklad, Amy, Nations, Sharon, Trivedi, Jaya, Hopkins, Steve, Kazamel, Mohamed, Alsharabati, Mohammad, Lu, Liang, Mumfrey-Thomas, Sandi, Woodall, Amy, Richman, David, Butters, Janelle, Lindsay, Molly, Mozaffar, Tahseen, Cash, Tiyonnoh, Goyal, Namita, Roy, Gulmohor, Mathew, Veena, Maqsood, Fatima, Minton, Brian, Jones, H. James, Rosenfeld, Jeffrey, Garcia, Rebekah, Garcia, Sonia, Echevarria, Laura, Pulley, Michael, Aranke, Shachie, Berger, Alan Ross, Shah, Jaimin, Shabbir, Yasmeen, Smith, Lisa, Varghese, Mary, Gutmann, Laurie, Gutmann, Ludwig, Swenson, Andrea, Olalde, Heena, Hafer-Macko, Charlene, Kwan, Justin, Zilliox, Lindsay, Callison, Karen, DiSanzo, Beth, Naunton, Kerry, Bilsker, Martin, Sharma, Khema, Reyes, Eliana, Cooley, Anne, Michon, Sara-Claude, Steele, Julie, Karam, Chafic Karam, Chopra, Manisha, Bird, Shawn, Kaufman, Jacob, Gallatti, Nichole, Vu, Tuan, Katzin, Lara, McClain, Terry, Harvey, Brittany, Hart, Adam, Huynh, Kristin, Beydoun, Said, Chilingaryan, Amaiak, Droker, Brian, Lin, Frank, Shah, Akshay, Tran, Anh, Akhter, Salma, Malekniazi, Ali, Tandan, Rup, Hehir, Michael, Waheed, Waqar, Lucy, Shannon, Weiss, Michael, Distad, Jane, Downing, Sharon, Strom, Susan, Lisak, Robert, Bernitsas, Evanthia, Khan, Omar, Kumar Sriwastava, Shitiz, Tselis, Alexandros, Jia, Kelly, Bertorini, Tulio, Arnold, Thomas, Henderson, Kendrick, Pillai, Rekha, Liu, Ye, Wheeler, Lauren, Hewlett, Jasmine, Vanderhook, Mollie, Dicapua, Daniel, Keung, Benison, Kumar, Aditya, Patwa, Huned, Robeson, Kimberly, Nye, Joan, Vu, Hong, Howard, James F, Jr, Utsugisawa, Kimiaki, Benatar, Michael, Murai, Hiroyuki, Barohn, Richard J, Illa, Isabel, Jacob, Saiju, Vissing, John, Burns, Ted M, Kissel, John T, Muppidi, Srikanth, Nowak, Richard J, O'Brien, Fanny, Wang, Jing-Jing, and Mantegazza, Renato

Published
2017
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7. Estudio epidemiológico multicéntrico sobre esclerosis lateral amiotrófica en la Ciudad de Buenos Aires

Author

Pérez Akly, Manuel, Schiava, Marianela, Melcom, Mario, Rodríguez, Gabriel, Gargiulo, Gisella, Bettini, Mariela, Reisin, Ricardo, Bendersky, Mariana, Barroso, Fabio, Brand, Patricio, de Ambrosi, Bruno, Di Egidio, Marianna, Fiorotto, Luis, Jáuregui, Agustín, Landriscina, Paula, Marchesoni, Cintia, Mazia, Claudio, Rey, Roberto, Rugiero, Marcelo, Salutto, Valeria, Tillard, Belén, and Fulgenzi, Ernesto

Published
2017
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8. Consenso argentino para el diagnóstico, seguimiento y tratamiento de la enfermedad de Pompe

Author

Dubrovsky, Alberto, Fulgenzi, Ernesto, Amartino, Hernán, Carlés, Daniel, Corderi, José, de Vito, Eduardo, Fainboim, Alejandro, Ferradás, Nélida, Guelbert, Norberto, Lubieniecki, Fabiana, Mazia, Claudio, Mesa, Lilia, Monges, Soledad, Pesquero, Joao, Reisin, Ricardo, Rugiero, Marcelo, Schenone, Andrea, Szlago, Marina, Taratuto, Ana Lia, and Zgaga, Marisa

Published
2014
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9. Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab:subgroup analysis of REGAIN and its extension study

Author

Siddiqi, Zaeem A., Nowak, Richard J., Mozaffar, Tahseen, O'Brien, Fanny, Yountz, Marcus, Patti, Francesco, Mazia, Claudio Gabriel, Wilken, Miguel, Barroso, Fabio, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Chaves, Marcelo, Vidal, Gonzalo, Garcia, Alejandra Dalila, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, Mercelis, Rudy, Mahieu, Délphine, Wagemaekers, Linda, Van Damme, Philip, Depreitere, Annelies, Schotte, Caroline, Smetcoren, Charlotte, Stevens, Olivier, Van Daele, Sien, Vandenbussche, Nicolas, Vanhee, Annelies, Verjans, Sarah, Vynckier, Jan, D'Hont, Ann, Tilkin, Petra, de Siqueira Carvalho, Alzira Alves, Brockhausen, Igor Dias, Feder, David, Ambrosio, Daniel, César, Pamela, Melo, Ana Paula, Ribeiro, Renata Martins, Rocha, Rosana, Rosa, Bruno Bezerra, Andersen, Henning, Vissing, John, Højgaard, Joan, Witting, Nanna, Pedersen, Jane, Weiss, Michael, Siddiqi, Zaeem A., Nowak, Richard J., Mozaffar, Tahseen, O'Brien, Fanny, Yountz, Marcus, Patti, Francesco, Mazia, Claudio Gabriel, Wilken, Miguel, Barroso, Fabio, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Chaves, Marcelo, Vidal, Gonzalo, Garcia, Alejandra Dalila, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, Mercelis, Rudy, Mahieu, Délphine, Wagemaekers, Linda, Van Damme, Philip, Depreitere, Annelies, Schotte, Caroline, Smetcoren, Charlotte, Stevens, Olivier, Van Daele, Sien, Vandenbussche, Nicolas, Vanhee, Annelies, Verjans, Sarah, Vynckier, Jan, D'Hont, Ann, Tilkin, Petra, de Siqueira Carvalho, Alzira Alves, Brockhausen, Igor Dias, Feder, David, Ambrosio, Daniel, César, Pamela, Melo, Ana Paula, Ribeiro, Renata Martins, Rocha, Rosana, Rosa, Bruno Bezerra, Andersen, Henning, Vissing, John, Højgaard, Joan, Witting, Nanna, Pedersen, Jane, and Weiss, Michael

Abstract

Introduction/Aims: Individuals with refractory generalized myasthenia gravis (gMG) who have a history of rituximab use and experience persistent symptoms represent a population with unmet treatment needs. The aim of this analysis was to evaluate the efficacy and safety of eculizumab in patients with refractory anti-acetylcholine receptor antibody-positive (AChR+) gMG previously treated with rituximab. Methods: This post hoc subgroup analysis of the phase 3 REGAIN study (NCT01997229) and its open-label extension (OLE; NCT02301624) compared baseline characteristics, safety, and response to eculizumab in participants who had previously received rituximab with those who had not. Rituximab use was not permitted within the 6 months before screening or during REGAIN/OLE. Results: Of 125 REGAIN participants, 14 had received rituximab previously (7 received placebo and 7 received eculizumab). In the previous-rituximab group, 57% had used at least four other immunosuppressants compared with 16% in the no-previous-rituximab group. Myasthenia Gravis Activities of Daily Living total scores from eculizumab baseline to week 130 of eculizumab treatment improved in both the previous-rituximab and no-previous-rituximab groups (least-squares mean −4.4, standard error of the mean [SEM] 1.0 [n = 9] and least-squares mean −4.6, SEM 0.3 [n = 67], respectively; difference = 0.2, 95% confidence interval −1.88 to 2.22). In addition, in both groups, most patients who were treated with eculizumab for 130 weeks achieved a Myasthenia Gravis Foundation of America post-intervention status of minimal manifestations (66.7% and 65.0%, respectively). The eculizumab safety profile was similar between groups and consistent with its established profile. Discussion: Eculizumab is an effective therapy for patients with refractory AChR+ gMG, irrespective of whether they had received rituximab treatment previously.

Published
2021

11. ‘Minimal symptom expression’ in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab

Author

Vissing, John, Jacob, Saiju, Fujita, Kenji P., O’Brien, Fanny, Howard, James F., Mazia, Claudio Gabriel, Wilken, Miguel, Barroso, Fabio, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Chaves, Marcelo, Vidal, Gonzalo, Garcia, Alejandra Dalila, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, Mercelis, Rudy, Mahieu, Délphine, Wagemaekers, Linda, Van Damme, Philip, Depreitere, Annelies, Schotte, Caroline, Smetcoren, Charlotte, Stevens, Olivier, Van Daele, Sien, Vandenbussche, Nicolas, Vanhee, Annelies, Verjans, Sarah, Vynckier, Jan, D’Hont, Ann, Tilkin, Petra, Alves de Siqueira Carvalho, Alzira, Dias Brockhausen, Igor, Feder, David, Ambrosio, Daniel, César, Pamela, Melo, Ana Paula, Martins Ribeiro, Renata, Rocha, Rosana, Rosa, Bruno Bezerra, Veiga, Thabata, da Silva, Luiz Augusto, Andersen, Henning, Harbo, Thomas, Vinge, Lotte, Krogh, Susanne, Mogensen, Anita, Højgaard, Joan, Witting, Nanna, Autzen, Anne Mette Ostergaard, Pedersen, Jane, Weiss, Michael, Vissing, John, Jacob, Saiju, Fujita, Kenji P., O’Brien, Fanny, Howard, James F., Mazia, Claudio Gabriel, Wilken, Miguel, Barroso, Fabio, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Chaves, Marcelo, Vidal, Gonzalo, Garcia, Alejandra Dalila, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, Mercelis, Rudy, Mahieu, Délphine, Wagemaekers, Linda, Van Damme, Philip, Depreitere, Annelies, Schotte, Caroline, Smetcoren, Charlotte, Stevens, Olivier, Van Daele, Sien, Vandenbussche, Nicolas, Vanhee, Annelies, Verjans, Sarah, Vynckier, Jan, D’Hont, Ann, Tilkin, Petra, Alves de Siqueira Carvalho, Alzira, Dias Brockhausen, Igor, Feder, David, Ambrosio, Daniel, César, Pamela, Melo, Ana Paula, Martins Ribeiro, Renata, Rocha, Rosana, Rosa, Bruno Bezerra, Veiga, Thabata, da Silva, Luiz Augusto, Andersen, Henning, Harbo, Thomas, Vinge, Lotte, Krogh, Susanne, Mogensen, Anita, Højgaard, Joan, Witting, Nanna, Autzen, Anne Mette Ostergaard, Pedersen, Jane, and Weiss, Michael

Abstract

Background: The efficacy and tolerability of eculizumab were assessed in REGAIN, a 26-week, phase 3, randomized, double-blind, placebo-controlled study in anti-acetylcholine receptor antibody-positive (AChR+) refractory generalized myasthenia gravis (gMG), and its open-label extension. Methods: Attainment of ‘minimal symptom expression’ was evaluated using patient-reported outcome measures of gMG symptoms [MG activities of daily living scale (MG-ADL), 15-item MG quality of life questionnaire (MG-QOL15)] at the completion of REGAIN and during the open-label extension. ‘Minimal symptom expression’ was defined as MG-ADL total score of 0–1 or MG-QOL15 total score of 0–3. Results: At REGAIN week 26, more eculizumab-treated patients achieved ‘minimal symptom expression’ versus placebo [MG-ADL: 21.4% vs 1.7%; difference 19.8%; 95% confidence interval (CI) 8.5, 31.0; p = 0.0007; MG-QOL15: 16.1% vs 1.7%; difference 14.4%; 95% CI 4.3, 24.6; p = 0.0069]. During the open-label extension, the proportion of patients in the placebo/eculizumab group who achieved ‘minimal symptom expression’ increased after initiating eculizumab treatment and was sustained through 130 weeks of open-label eculizumab (MG-ADL: 1.7 to 27.8%; MG-QOL15: 1.7 to 19.4%). At extension study week 130, similar proportions of patients in the eculizumab/eculizumab and placebo/eculizumab groups achieved ‘minimal symptom expression’ (MG-ADL: 22.9% and 27.8%, respectively, p = 0.7861; MG-QOL15: 14.3% and 19.4%, respectively, p = 0.7531). The long-term tolerability of eculizumab was consistent with previous reports. Conclusions: Patients with AChR+ refractory gMG who receive eculizumab can achieve sustained ‘minimal symptom expression’ based on patient-reported outcomes. ‘Minimal symptom expression’ may be a useful tool in measuring therapy effectiveness in gMG. Trial registration: ClinicalTrials.gov NCT01997229, NCT02301624.

Published
2020

12. Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study.

Author

Siddiqi, Zaeem A., Nowak, Richard J., Mozaffar, Tahseen, O'Brien, Fanny, Yountz, Marcus, Patti, Francesco, Mazia, Claudio Gabriel, Wilken, Miguel, Barroso, Fabio, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Chaves, Marcelo, Vidal, Gonzalo, Garcia, Alejandra Dalila, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, and Mercelis, Rudy

Abstract

Introduction/Aims: Individuals with refractory generalized myasthenia gravis (gMG) who have a history of rituximab use and experience persistent symptoms represent a population with unmet treatment needs. The aim of this analysis was to evaluate the efficacy and safety of eculizumab in patients with refractory anti‐acetylcholine receptor antibody‐positive (AChR+) gMG previously treated with rituximab. Methods: This post hoc subgroup analysis of the phase 3 REGAIN study (NCT01997229) and its open‐label extension (OLE; NCT02301624) compared baseline characteristics, safety, and response to eculizumab in participants who had previously received rituximab with those who had not. Rituximab use was not permitted within the 6 months before screening or during REGAIN/OLE. Results: Of 125 REGAIN participants, 14 had received rituximab previously (7 received placebo and 7 received eculizumab). In the previous‐rituximab group, 57% had used at least four other immunosuppressants compared with 16% in the no‐previous‐rituximab group. Myasthenia Gravis Activities of Daily Living total scores from eculizumab baseline to week 130 of eculizumab treatment improved in both the previous‐rituximab and no‐previous‐rituximab groups (least‐squares mean −4.4, standard error of the mean [SEM] 1.0 [n = 9] and least‐squares mean −4.6, SEM 0.3 [n = 67], respectively; difference = 0.2, 95% confidence interval −1.88 to 2.22). In addition, in both groups, most patients who were treated with eculizumab for 130 weeks achieved a Myasthenia Gravis Foundation of America post‐intervention status of minimal manifestations (66.7% and 65.0%, respectively). The eculizumab safety profile was similar between groups and consistent with its established profile. Discussion: Eculizumab is an effective therapy for patients with refractory AChR+ gMG, irrespective of whether they had received rituximab treatment previously. [ABSTRACT FROM AUTHOR]

Published
2021
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13. Long-term safety and efficacy of eculizumab in generalized myasthenia gravis

Author

Muppidi, Srikanth, Utsugisawa, Kimiaki, Benatar, Michael, Murai, Hiroyuki, Barohn, Richard J., Illa, Isabel, Jacob, Saiju, Vissing, John, Burns, Ted M., Kissel, John T., Nowak, Richard J., Andersen, Henning, Casasnovas, Carlos, de Bleecker, Jan L., Vu, Tuan H., Mantegazza, Renato, O'Brien, Fanny L., Wang, Jing Jing, Fujita, Kenji P., Howard, James F., Mazia, Claudio Gabriel, Wilken, Miguel, Barroso, Fabio, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Chaves, Marcelo, Vidal, Gonzalo, Garcia, Alejandra Dalila, van den Abeele, Guy, de Koning, Kathy, de Mey, Katrien, Mercelis, Rudy, Mahieu, D. lphine, Wagemaekers, Linda, van Damme, Philip, Depreitere, Annelies, Schotte, Caroline, Smetcoren, Charlotte, Stevens, Olivier, van Daele, Sien, Vandenbussche, Nicolas, Vanhee, Annelies, Verjans, Sarah, Vynckier, Jan, D'Hondt, Ann, Tilkin, Petra, Alves de Siqueira Carvalho, Alzira, Dias Brockhausen, Igor, Feder, David, Ambrosio, Daniel, César, Pamela, Melo, Ana Paula, Martins Ribeiro, Renata, Rocha, Rosana, Bezerra Rosa, Bruno, Veiga, Thabata, da Silva, Luiz Augusto, Santos Engel, Murilo, Gonçalves Geraldo, Jordana, Ananias Morita, Maria da Penha, Nogueira Coelho, Erica, Paiva, Gabriel, Pozo, Marina, Prando, Natalia, Martineli Torres, Debora Dada, Butinhao, Cristiani Fernanda, Duran, Gustavo, Gomes da Silva, Tamires Cristina, Otavio Maia Gonçalves, Luiz, Pazetto, Lucas Eduardo, Fialho, Tomás Augusto Suriane, Renata Cubas Volpe, Luciana, Souza Duca, Luciana, Gheller Friedrich, Maurício André, Guerreiro, Alexandre, Mohr, Henrique, Pereira Martins, Maurer, da Cruz Pacheco, Daiane, Ferreira, Luciana, Macagnan, Ana Paula, Pinto, Graziela, de Cassia Santos, Aline, Souza Bulle Oliveira, Acary, Amaral Andrade, Ana Carolina, Annes, Marcelo, Duarte Silva, Liene, Cavalcante Lino, Valeria, Pinto, Wladimir, Assis, Natália, Carrara, Fernanda, Miranda, Carolina, Souza, Iandra, Fernandes, Patricia, Siddiqi, Zaeem, Phan, Cecile, Narayan, Jeffrey, Blackmore, Derrick, Mallon, Ashley, Roderus, Rikki, Watt, Elizabeth, Vohanka, Stanislav, Bednarik, Josef, Chmelikova, Magda, Cierny, Marek, Toncrova, Stanislava, Junkerova, Jana, Kurkova, Barbora, Reguliova, Katarina, Zapletalova, Olga, Pitha, Jiri, Novakova, Iveta, Tyblova, Michaela, Jurajdova, Ivana, Wolfova, Marcela, Harbo, Thomas, Vinge, Lotte, Krogh, Susanne, Mogensen, Anita, Højgaard, Joan, Witting, Nanna, Ostergaard Autzen, Anne, Pedersen, Jane, Eralinna, Juha-Pekka, Laaksonen, Mikko, Oksaranta, Olli, Harrison, Tuula, Eriksson, Jaana, Rozsa, Csilla, Horvath, Melinda, Lovas, Gabor, Matolcsi, Judit, Szabo, Gyorgyi, Jakab, Gedeonne, Szabadosne, Brigitta, Vecsei, Laszlo, Dezsi, Livia, Varga, Edina, Konyane, Monika, Antonini, Giovanni, di Pasquale, Antonella, Garibaldi, Matteo, Morino, Stefania, Troili, Fernanda, Fionda, Laura, Filla, Allessandro, Costabile, Teresa, Marano, Enrico, Saccà, Francesco, Fasanaro, Angiola, Marsili, Angela, Puorro, Giorgia, Antozzi, Carlo, Bonanno, Silvia, Camera, Giorgia, Locatelli, Alberta, Maggi, Lorenzo, Pasanisi, Maria, Campanella, Angela, Evoli, Amelia, Alboini, Paolo Emilio, D'Amato, Valentina, Iorio, Raffaele, Inghilleri, Maurizio, Frasca, Vittorio, Giacomelli, Elena, Gori, Maria, Lopergolo, Diego, Onesti, Emanuela, Gabriele, Maria, Uzawa, Akiyuki, Kanai, Tetsuya, Kawaguchi, Naoki, Mori, Masahiro, Kaneko, Yoko, Kanzaki, Akiko, Kobayashi, Eri, Masaki, Katsuhisa, Matsuse, Dai, Matsushita, Takuya, Uehara, Taira, Shimpo, Misa, Jingu, Maki, Kikutake, Keiko, Nakamura, Yumiko, Sano, Yoshiko, Nagane, Yuriko, Kamegamori, Ikuko, Tsuda, Tomoko, Fujii, Yuko, Futono, Kazumi, Ozawa, Yukiko, Mizugami, Aya, Saito, Yuka, Suzuki, Hidekazu, Morikawa, Miyuki, Samukawa, Makoto, Kamakura, Sachiko, Miyawaki, Eriko, Shiraishi, Hirokazu, Mitazaki, Teiichiro, Motomura, Masakatsu, Mukaino, Akihiro, Yoshimura, Shunsuke, Asada, Shizuka, Yoshida, Seiko, Amamoto, Shoko, Kobashikawa, Tomomi, Koga, Megumi, Maeda, Yasuko, Takada, Kazumi, Takada, Mihoko, Tsurumaru, Masako, Yamashita, Yumi, Suzuki, Yasushi, Akiyama, Tetsuya, Narikawa, Koichi, Tano, Ohito, Tsukita, Kenichi, Kurihara, Rikako, Meguro, Fumie, Fukuda, Yusuke, Sato, Miwako, Okumura, Meinoshin, Funaka, Soichiro, Kawamura, Tomohiro, Makamori, Masayuki, Takahashi, Masanori, Taichi, Namie, Hasuike, Tomoya, Higuchi, Eriko, Kobayashi, Hisako, Osakada, Kaori, Imai, Tomihiro, Tsuda, Emiko, Shimohama, Shun, Hayashi, Takashi, Hisahara, Shin, Kawamata, Jun, Murahara, Takashi, Saitoh, Masaki, Suzuki, Shuichiro, Yamamoto, Daisuke, Ishiyama, Yoko, Ishiyama, Naoko, Noshiro, Mayuko, Takeyama, Rumi, Uwasa, Kaori, Yasuda, Ikuko, van der Kooi, Anneke, de Visser, Marianne, Gibson, Tamar, Kim, Byung-Jo, Lee, Chang Nyoung, Koo, Yong Seo, Seok, Hung Youl, Kang, Hoo Nam, Ra, HyeJin, Kim, Byoung Joon, Cho, Eun Bin, Choi, MiSong, Lee, HyeLim, Min, Ju-Hong, Seok, Jinmyoung, Lee, JiEun, Koh, Da Yoon, Kwon, JuYoung, Park, SangAe, Choi, Eun Hwa, Hong, Yoon-Ho, Ahn, So-Hyun, Koo, Dae Lim, Lim, Jae-Sung, Shin, Chae Won, Hwang, Ji Ye, Kim, Miri, Kim, Seung Min, Jeong, Ha-Neul, Jung, JinWoo, Kim, Yool-hee, Lee, Hyung Seok, Shin, Ha Young, Hwang, Eun Bi, Shin, Miju, Alberti Aguilo, Maria Antonia, Homedes-Pedret, Christian, Julia Palacios, Natalia, Diez Porras, Laura, Velez Santamaria, Valentina, Lazaro, Ana, Diez Tejedor, Exuperio, Gomez Salcedo, Pilar, Fernandez-Fournier, Mireya, Lopez Ruiz, Pedro, Rodriguez de Rivera, Francisco Javier, Sastre, Maria, Gamez, Josep, Sune, Pilar, Salvado, Maria, Gili, Gisela, Mazuela, Gonzalo, Cortes Vicente, Elena, Diaz-Manera, Jordi, Querol Gutierrez, Luis Antonio, Rojas Garcia, Ricardo, Vidal, Nuria, Arribas-Ibar, Elisabet, Piehl, Fredrik, Hietala, Albert, Bjarbo, Lena, Sengun, Ihsan, Meherremova, Arzu, Ozcelik, Pinar, Balkan, Bengu, Tuga, Celal, Ugur, Muzeyyen, Erdem-Ozdamar, Sevim, Bekircan-Kurt, Can Ebru, Acar, Nazire Pinar, Yilmaz, Ezgi, Caliskan, Yagmur, Orsel, Gulsah, Efendi, Husnu, Aydinlik, Seda, Cavus, Hakan, Kutlu, Ayse, Becerikli, Gulsar, Semiz, Cansu, Tun, Ozlem, Terzi, Murat, Dogan, Baki, Onar, Musa Kazim, Sen, Sedat, Kirbas Cavdar, Tugce, Veske, Adife, Norwood, Fiona, Dimitriou, Aikaterini, Gollogly, Jakit, Mahdi-Rogers, Mohamed, Seddigh, Arshira, Sokratous, Giannis, Maier, Gal, Sohail, Faisal, Sadalage, Girija, Torane, Pravin, Brown, Claire, Shah, Amna, Sathasivam, Sivakumar, Arndt, Heike, Davies, Debbie, Watling, Dave, Amato, Anthony, Cochrane, Thomas, Salajegheh, Mohammed, Roe, Kristen, Amato, Katherine, Toska, Shirli, Wolfe, Gil, Silvestri, Nicholas, Patrick, Kara, Zakalik, Karen, Katz, Jonathan, Miller, Robert, Engel, Marguerite, Forshew, Dallas, Bravver, Elena, Brooks, Benjamin, Plevka, Sarah, Burdette, Maryanne, Cunningham, Scott, Sanjak, Mohammad, Kramer, Megan, Nemeth, Joanne, Schommer, Clara, Tierney, Scott, Juel, Vern, Guptill, Jeffrey, Hobson-Webb, Lisa, Massey, Janice, Beck, Kate, Carnes, Donna, Loor, John, Anderson, Amanda, Pascuzzi, Robert, Bodkin, Cynthia, Kincaid, John, Snook, Riley, Guingrich, Sandra, Micheels, Angela, Chaudhry, Vinay, Corse, Andrea, Mosmiller, Betsy, Kelley, Andrea, Ho, Doreen, Srinivasan, Jayashri, Vytopil, Michal, Jara, Jordan, Ventura, Nicholas, Scala, Stephanie, Carter, Cynthia, Donahue, Craig, Herbert, Carol, Weiner, Elaine, Alam, Sharmeen, McKinnon, Jonathan, Haar, Laura, McKinnon, Naya, Alcon, Karan, McKenna, Kaitlyn, Sattar, Nadia, Daniels, Kevin, Jeffery, Dennis, Freimer, Miriam, Hoyle, Joseph Chad, Agriesti, Julie, Chelnick, Sharon, Mezache, Louisa, Pineda, Colleen, Muharrem, Filiz, Karam, Chafic, Khoury, Julie, Marburger, Tessa, Kaur, Harpreet, Dimitrova, Diana, Gilchrist, James, Agrawal, Brajesh, Elsayed, Mona, Kohlrus, Stephanie, Andoin, Angela, Darnell, Taylor, Golden, Laura, Lokaitis, Barbara, Seelback, Jenna, Goyal, Neelam, Sakamuri, Sarada, So, Yuen T., Paulose, Shirley, Pol, Sabrina, Welsh, Lesly, Bhavaraju-Sanka, Ratna, Tobon Gonzales, Alejandro, Dishman, Lorraine, Jones, Floyd, Gonzalez, Anna, Padilla, Patricia, Saklad, Amy, Silva, Marcela, Nations, Sharon, Trivedi, Jaya, Hopkins, Steve, Kazamel, Mohamed, Alsharabati, Mohammad, Lu, Liang, Nozaki, Kenkichi, Mumfrey-Thomas, Sandi, Woodall, Amy, Mozaffar, Tahseen, Cash, Tiyonnoh, Goyal, Namita, Roy, Gulmohor, Mathew, Veena, Maqsood, Fatima, Minton, Brian, Jones, H. James, Rosenfeld, Jeffrey, Garcia, Rebekah, Echevarria, Laura, Garcia, Sonia, Pulley, Michael, Aranke, Shachie, Berger, Alan Ross, Shah, Jaimin, Shabbir, Yasmeen, Smith, Lisa, Varghese, Mary, Gutmann, Laurie, Gutmann, Ludwig, Jerath, Nivedita, Nance, Christopher, Swenson, Andrea, Olalde, Heena, Kressin, Nicole, Sieren, Jeri, Dimachkie, Mazen, Glenn, Melanie, McVey, April, Pasnoor, Mamatha, Statland, Jeffery, Wang, Yunxia, Liu, Tina, Emmons, Kelley, Jenci, Nicole, Locheke, Jerry, Fondaw, Alex, Johns, Kathryn, Rico, Gabrielle, Walsh, Maureen, Herbelin, Laura, Hafer-Macko, Charlene, Kwan, Justin, Zilliox, Lindsay, Callison, Karen, Young, Valerie, DiSanzo, Beth, Naunton, Kerry, Bilsker, Martin, Sharma, Khema, Cooley, Anne, Reyes, Eliana, Michon, Sara-Claude, Sheldon, Danielle, Steele, Julie, Chopra, Manisha, Traub, Rebecca, Katzin, Lara, McClain, Terry, Harvey, Brittany, Hart, Adam, Huynh, Kristin, Beydoun, Said, Chilingaryan, Amaiak, Doan, Victor, Droker, Brian, Gong, Hui, Karimi, Sanaz, Lin, Frank, Pokala, Krishna, Shah, Akshay, Tran, Anh, Akhter, Salma, Malekniazi, Ali, Tandan, Rup, Hehir, Michael, Waheed, Waqar, Lucy, Shannon, Weiss, Michael, Distad, Jane, Strom, Susan, Downing, Sharon, Kim, Bryan, Bertorini, Tulio, Arnold, Thomas, Hendersen, Kendrick, Pillai, Rekha, Liu, Ye, Wheeler, Lauren, Hewlett, Jasmine, Vanderhook, Mollie, Dicapua, Daniel, Keung, Benison, Kumar, Aditya, Patwa, Huned, Robeson, Kimberly, Yang, Irene, Nye, Joan, Vu, Hong, Muppidi, S., Utsugisawa, K., Benatar, M., Murai, H., Barohn, R. J., Illa, I., Jacob, S., Vissing, J., Burns, T. M., Kissel, J. T., Nowak, R. J., Andersen, H., Casasnovas, C., de Bleecker, J. L., Vu, T. H., Mantegazza, R., O'Brien, F. L., Wang, J. J., Fujita, K. P., Howard, J. F., Mazia, C. G., Wilken, M., Barroso, F., Saba, J., Rugiero, M., Bettini, M., Chaves, M., Vidal, G., Garcia, A. D., Van den Abeele, G., de Koning, K., De Mey, K., Mercelis, R., Mahieu, D., Wagemaekers, L., Van Damme, P., Depreitere, A., Schotte, C., Smetcoren, C., Stevens, O., Van Daele, S., Vandenbussche, N., Vanhee, A., Verjans, S., Vynckier, J., D'Hondt, A., Tilkin, P., Alves de Siqueira Carvalho, A., Dias Brockhausen, I., Feder, D., Ambrosio, D., Cesar, P., Melo, A. P., Martins Ribeiro, R., Rocha, R., Bezerra Rosa, B., Veiga, T., da Silva, L. A., Santos Engel, M., Goncalves Geraldo, J., Ananias Morita, M. D. P., Nogueira Coelho, E., Paiva, G., Pozo, M., Prando, N., Martineli Torres, D. D., Butinhao, C. F., Duran, G., Gomes da Silva, T. C., Otavio Maia Goncalves, L., Pazetto, L. E., Fialho, T. A. S., Renata Cubas Volpe, L., Souza Duca, L., Gheller Friedrich, M. A., Guerreiro, A., Mohr, H., Pereira Martins, M., da Cruz Pacheco, D., Ferreira, L., Macagnan, A. P., Pinto, G., de Cassia Santos, A., Souza Bulle Oliveira, A., Amaral Andrade, A. C., Annes, M., Duarte Silva, L., Cavalcante Lino, V., Pinto, W., Assis, N., Carrara, F., Miranda, C., Souza, I., Fernandes, P., Siddiqi, Z., Phan, C., Narayan, J., Blackmore, D., Mallon, A., Roderus, R., Watt, E., Vohanka, S., Bednarik, J., Chmelikova, M., Cierny, M., Toncrova, S., Junkerova, J., Kurkova, B., Reguliova, K., Zapletalova, O., Pitha, J., Novakova, I., Tyblova, M., Jurajdova, I., Wolfova, M., Harbo, T., Vinge, L., Krogh, S., Mogensen, A., Hojgaard, J., Witting, N., Ostergaard Autzen, A., Pedersen, J., Eralinna, J. -P., Laaksonen, M., Oksaranta, O., Harrison, T., Eriksson, J., Rozsa, C., Horvath, M., Lovas, G., Matolcsi, J., Szabo, G., Jakab, G., Szabadosne, B., Vecsei, L., Dezsi, L., Varga, E., Konyane, M., Antonini, G., Di Pasquale, A., Garibaldi, M., Morino, S., Troili, F., Fionda, L., Filla, A., Costabile, T., Marano, E., Sacca, F., Fasanaro, A., Marsili, A., Puorro, G., Antozzi, C., Bonanno, S., Camera, G., Locatelli, A., Maggi, L., Pasanisi, M., Campanella, A., Evoli, A., Alboini, P. E., D'Amato, V., Iorio, R., Inghilleri, M., Frasca, V., Giacomelli, E., Gori, M., Lopergolo, D., Onesti, E., Gabriele, M., Uzawa, A., Kanai, T., Kawaguchi, N., Mori, M., Kaneko, Y., Kanzaki, A., Kobayashi, E., Masaki, K., Matsuse, D., Matsushita, T., Uehara, T., Shimpo, M., Jingu, M., Kikutake, K., Nakamura, Y., Sano, Y., Nagane, Y., Kamegamori, I., Tsuda, T., Fujii, Y., Futono, K., Ozawa, Y., Mizugami, A., Saito, Y., Suzuki, H., Morikawa, M., Samukawa, M., Kamakura, S., Miyawaki, E., Shiraishi, H., Mitazaki, T., Motomura, M., Mukaino, A., Yoshimura, S., Asada, S., Yoshida, S., Amamoto, S., Kobashikawa, T., Koga, M., Maeda, Y., Takada, K., Takada, M., Tsurumaru, M., Yamashita, Y., Suzuki, Y., Akiyama, T., Narikawa, K., Tano, O., Tsukita, K., Kurihara, R., Meguro, F., Fukuda, Y., Sato, M., Okumura, M., Funaka, S., Kawamura, T., Makamori, M., Takahashi, M., Taichi, N., Hasuike, T., Higuchi, E., Kobayashi, H., Osakada, K., Imai, T., Tsuda, E., Shimohama, S., Hayashi, T., Hisahara, S., Kawamata, J., Murahara, T., Saitoh, M., Suzuki, S., Yamamoto, D., Ishiyama, Y., Ishiyama, N., Noshiro, M., Takeyama, R., Uwasa, K., Yasuda, I., van der Kooi, A., de Visser, M., Gibson, T., Kim, B. -J., Lee, C. N., Koo, Y. S., Seok, H. Y., Kang, H. N., Ra, H., Kim, B. J., Cho, E. B., Choi, M., Lee, H., Min, J. -H., Seok, J., Lee, J., Koh, D. Y., Kwon, J., Park, S., Choi, E. H., Hong, Y. -H., Ahn, S. -H., Koo, D. L., Lim, J. -S., Shin, C. W., Hwang, J. Y., Kim, M., Kim, S. M., Jeong, H. -N., Jung, J., Kim, Y. -H., Lee, H. S., Shin, H. Y., Hwang, E. B., Shin, M., Alberti Aguilo, M. A., Homedes-Pedret, C., Julia Palacios, N., Diez Porras, L., Velez Santamaria, V., Lazaro, A., Diez Tejedor, E., Gomez Salcedo, P., Fernandez-Fournier, M., Lopez Ruiz, P., Rodriguez de Rivera, F. J., Sastre, M., Gamez, J., Sune, P., Salvado, M., Gili, G., Mazuela, G., Cortes Vicente, E., Diaz-Manera, J., Querol Gutierrez, L. A., Rojas Garcia, R., Vidal, N., Arribas-Ibar, E., Piehl, F., Hietala, A., Bjarbo, L., Sengun, I., Meherremova, A., Ozcelik, P., Balkan, B., Tuga, C., Ugur, M., Erdem-Ozdamar, S., Bekircan-Kurt, C. E., Acar, N. P., Yilmaz, E., Caliskan, Y., Orsel, G., Efendi, H., Aydinlik, S., Cavus, H., Kutlu, A., Becerikli, G., Semiz, C., Tun, O., Terzi, M., Dogan, B., Onar, M. K., Sen, S., Kirbas Cavdar, T., Veske, A., Norwood, F., Dimitriou, A., Gollogly, J., Mahdi-Rogers, M., Seddigh, A., Sokratous, G., Maier, G., Sohail, F., Sadalage, G., Torane, P., Brown, C., Shah, A., Sathasivam, S., Arndt, H., Davies, D., Watling, D., Amato, A., Cochrane, T., Salajegheh, M., Roe, K., Amato, K., Toska, S., Wolfe, G., Silvestri, N., Patrick, K., Zakalik, K., Katz, J., Miller, R., Engel, M., Forshew, D., Bravver, E., Brooks, B., Plevka, S., Burdette, M., Cunningham, S., Sanjak, M., Kramer, M., Nemeth, J., Schommer, C., Tierney, S., Juel, V., Guptill, J., Hobson-Webb, L., Massey, J., Beck, K., Carnes, D., Loor, J., Anderson, A., Pascuzzi, R., Bodkin, C., Kincaid, J., Snook, R., Guingrich, S., Micheels, A., Chaudhry, V., Corse, A., Mosmiller, B., Kelley, A., Ho, D., Srinivasan, J., Vytopil, M., Jara, J., Ventura, N., Scala, S., Carter, C., Donahue, C., Herbert, C., Weiner, E., Alam, S., Mckinnon, J., Haar, L., Mckinnon, N., Alcon, K., Mckenna, K., Sattar, N., Daniels, K., Jeffery, D., Freimer, M., Hoyle, J. C., Agriesti, J., Chelnick, S., Mezache, L., Pineda, C., Muharrem, F., Karam, C., Khoury, J., Marburger, T., Kaur, H., Dimitrova, D., Gilchrist, J., Agrawal, B., Elsayed, M., Kohlrus, S., Andoin, A., Darnell, T., Golden, L., Lokaitis, B., Seelback, J., Goyal, N., Sakamuri, S., So, Y. T., Paulose, S., Pol, S., Welsh, L., Bhavaraju-Sanka, R., Tobon Gonzales, A., Dishman, L., Jones, F., Gonzalez, A., Padilla, P., Saklad, A., Silva, M., Nations, S., Trivedi, J., Hopkins, S., Kazamel, M., Alsharabati, M., Lu, L., Nozaki, K., Mumfrey-Thomas, S., Woodall, A., Mozaffar, T., Cash, T., Roy, G., Mathew, V., Maqsood, F., Minton, B., Jones, H. J., Rosenfeld, J., Garcia, R., Echevarria, L., Garcia, S., Pulley, M., Aranke, S., Berger, A. R., Shah, J., Shabbir, Y., Smith, L., Varghese, M., Gutmann, L., Jerath, N., Nance, C., Swenson, A., Olalde, H., Kressin, N., Sieren, J., Dimachkie, M., Glenn, M., Mcvey, A., Pasnoor, M., Statland, J., Wang, Y., Liu, T., Emmons, K., Jenci, N., Locheke, J., Fondaw, A., Johns, K., Rico, G., Walsh, M., Herbelin, L., Hafer-Macko, C., Kwan, J., Zilliox, L., Callison, K., Young, V., Disanzo, B., Naunton, K., Bilsker, M., Sharma, K., Cooley, A., Reyes, E., Michon, S. -C., Sheldon, D., Steele, J., Chopra, M., Traub, R., Katzin, L., Mcclain, T., Harvey, B., Hart, A., Huynh, K., Beydoun, S., Chilingaryan, A., Doan, V., Droker, B., Gong, H., Karimi, S., Lin, F., Pokala, K., Tran, A., Akhter, S., Malekniazi, A., Tandan, R., Hehir, M., Waheed, W., Lucy, S., Weiss, M., Distad, J., Strom, S., Downing, S., Kim, B., Bertorini, T., Arnold, T., Hendersen, K., Pillai, R., Liu, Y., Wheeler, L., Hewlett, J., Vanderhook, M., Dicapua, D., Keung, B., Kumar, A., Patwa, H., Robeson, K., Yang, I., Nye, J., Vu, H., Neurology, AII - Infectious diseases, ANS - Neuroinfection & -inflammation, APH - Methodology, and APH - Quality of Care

Subjects
0301 basic medicine, Male, Pediatrics, Exacerbation, Physiology, Heart Diseases/chemically induced, Myasthenia Gravis/drug therapy, 030105 genetics & heredity, THERAPY, DOUBLE-BLIND, 0302 clinical medicine, Quality of life, Monoclonal, Activities of Daily Living, Medicine and Health Sciences, Functional ability, Longitudinal Studies, Humanized, Angioedema/chemically induced, MG-ADL, QMG, Eculizumab, Middle Aged, myasthenia gravi, 3. Good health, Treatment Outcome, Antibodies, Monoclonal, Humanized/therapeutic use, Meningococcal Infections/epidemiology, Disease Progression, Female, eculizumab, Life Sciences & Biomedicine, COMPLEMENT INHIBITOR ECULIZUMAB, medicine.drug, Adult, medicine.medical_specialty, MG-QOL15, Heart Diseases, Clinical Neurology, Meningococcal Vaccines, Antibodies, Monoclonal, Humanized, Placebo, Antibodies, ACETYLCHOLINE-RECEPTOR, Complement Inactivating Agents/therapeutic use, 03 medical and health sciences, Cellular and Molecular Neuroscience, Refractory, Physiology (medical), Injection Site Reaction/epidemiology, Myasthenia Gravis, medicine, Aspergillosis, Aspergillosis/epidemiology, Humans, Muscle Strength, Angioedema, myasthenia gravis, Science & Technology, business.industry, MGC, Neurosciences, Meningococcal Vaccines/therapeutic use, medicine.disease, Interim analysis, Complement Inactivating Agents, Injection Site Reaction, Meningococcal Infections, Quality of Life, Myasthenia gravis, ANTIBODY, Neurosciences & Neurology, Neurology (clinical), business, 030217 neurology & neurosurgery
Abstract

INTRODUCTION: Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open-label extension of REGAIN, evaluating eculizumab's long-term safety and efficacy. METHODS: Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients. RESULTS: The safety profile of eculizumab was consistent with REGAIN; no cases of meningococcal infection were reported during the interim analysis period. Myasthenia gravis exacerbation rate was reduced by 75% from the year before REGAIN (P

Published
2019
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14. Response to eculizumab in patients with myasthenia gravis recently treated with chronic IVIg: a subgroup analysis of REGAIN and its open-label extension study

Author

Jacob, Saiju, primary, Murai, Hiroyuki, additional, Utsugisawa, Kimiaki, additional, Nowak, Richard J., additional, Wiendl, Heinz, additional, Fujita, Kenji P., additional, O’Brien, Fanny, additional, Howard, James F., additional, Mazia, Claudio Gabriel, additional, Wilken, Miguel, additional, Barroso, Fabio, additional, Saba, Juliet, additional, De Bleecker, Jan, additional, Van den Abeele, Guy, additional, de Koning, Kathy, additional, De Mey, Katrien, additional, Carvalho, Alzira Alves de Siqueira, additional, Brockhausen, Igor Dias, additional, Feder, David, additional, Ambrosio, Daniel, additional, César, Pamela, additional, Melo, Ana Paula, additional, Ribeiro, Renata Martins, additional, Rocha, Rosana, additional, Rosa, Bruno Bezerra, additional, Veiga, Thabata, additional, da Silva, Luiz Augusto, additional, Engel, Murilo Santos, additional, Gonçalves Geraldo, Jordana, additional, Nagane, Yuriko, additional, Kamegamori, Ikuko, additional, Tsuda, Tomoko, additional, Fujii, Yuko, additional, Futono, Kazumi, additional, Ozawa, Yukiko, additional, Mizugami, Aya, additional, Saito, Yuka, additional, van der Kooi, Anneke, additional, de Visser, Marianne, additional, Gibson, Tamar, additional, Kim, Seung Min, additional, Jeong, JinWoo, additional, Jung, Ha-Neul, additional, Kim, Yool-hee, additional, Lee, Hyung Seok, additional, Shin, Ha Young, additional, Hwang, Eun Bi, additional, Shin, Miju, additional, Carbonell, Josep Gamez, additional, Sune, Pilar, additional, Figueras, Maria Salvado, additional, Gili, Gisela, additional, Mazuela, Gonzalo, additional, Piehl, Fredrik, additional, Hietala, Albert, additional, Bjarbo, Lena, additional, Erdem-Ozdamar, Sevim, additional, Bekircan-Kurt, Can Ebru, additional, Acar, Nazire Pinar, additional, Yilmaz, Ezgi, additional, Caliskan, Yagmur, additional, Orsel, Gulsah, additional, Amato, Anthony, additional, Cochrane, Thomas, additional, Salajegheh, Mohammed, additional, Roe, Kristen, additional, Amato, Katherine, additional, Toska, Shirli, additional, McKinnon, Jonathan, additional, Haar, Laura, additional, McKinnon, Naya, additional, Alcon, Karan, additional, McKenna, Kaitlyn, additional, Sattar, Nadia, additional, Daniels, Kevin, additional, Jeffery, Dennis, additional, Mozaffar, Tahseen, additional, Cash, Tiyonnoh, additional, Goyal, Namita, additional, Roy, Gulmohor, additional, Mathew, Veena, additional, Maqsood, Fatima, additional, Minton, Brian, additional, Hafer-Macko, Charlene, additional, Kwan, Justin, additional, Zilliox, Lindsay, additional, Callison, Karen, additional, Young, Valerie, additional, DiSanzo, Beth, additional, Naunton, Kerry, additional, Vu, Tuan, additional, Katzin, Lara, additional, McClain, Terry, additional, Harvey, Brittany, additional, Hart, Adam, additional, Huynh, Kristin, additional, Beydoun, Said, additional, Chilingaryan, Amaiak, additional, Doan, Victor, additional, Droker, Brian, additional, Gong, Hui, additional, Karimi, Sanaz, additional, Lin, Frank, additional, Polaka, Krishna, additional, Shah, Akshay, additional, Tran, Anh, additional, Akhter, Salma, additional, Malekniazi, Ali, additional, Tandan, Rup, additional, Hehir, Michael, additional, Waheed, Waqar, additional, Lucy, Shannon, additional, Bertorini, Tulio, additional, Arnold, Thomas, additional, Hendersen, Kendrick, additional, Pillai, Rekha, additional, Liu, Ye, additional, Wheeler, Lauren, additional, Hewlett, Jasmine, additional, and Vanderhook, Mollie, additional

Published
2020
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15. Consenso argentino sobre enfermedad de Pompe de inicio tardío

Author

Dubrovsky, Alberto, Fulgenzi, Ernesto, De Vito, Eduardo L, Barroso, Fabio, Berardo, Andrés, Bettini, Mariela, Binaghi, Daniela, Calabrese, Esteban, Carlés, Daniel, Chaves, Marcelo, Chloca, Fernando, Conti, Eugenia, Corderi, Jose, Di Gennaro, Federico, Ferradás, Nélida, Jáuregui, Agustín, Lubieniecki, Fabiana, Mazia, Claudio, Medina, Marta, Pirra, Laura, Politei, Juan, Reisin, Ricardo, Rosa, Alberto L, Rugiero, Marcelo, Salutto, Valeria, Schenone, Andrea, Sussini, Mario, and Taratuto, Ana L

Subjects
Enfermedad de Pompe, Consensus, Consenso, Glucogenosis tipo II, Alfa-glucosidase, Type II glycogenosis, Enzyme replacement therapy, Acid lisosomal maltase deficiency, Alfa-glucosidasa ácida, Deficiencia de maltasa ácida lisosomal, Pompe’s disease, Terapia de reemplazo enzimático
Abstract

La enfermedad de Pompe (EP) es un desorden metabólico autosómico recesivo infrecuente, producido por la ausencia o deficiencia de la enzima lisosomal alfa-glucosidasa ácida en los tejidos de los individuos afectados. Se considera enfermedad de Pompe de inicio tardío (EPIT) en aquellos individuos de más de un año de edad al comienzo de los síntomas. El objetivo del presente consenso es el de actualizar las pautas y recomendaciones para un correcto tratamiento de los pacientes con EPIT, tomando como referencia los lineamientos del Consenso Argentino para el diagnóstico, seguimiento y tratamiento de la enfermedad de Pompe publicado en el año 2013. Se organizó un consenso que reunió profesionales con experiencia en la EP en las áreas de clínica médica, diagnóstico de laboratorio, neuropatología, neumonología, nutrición, neurología, enfermedades metabólicas, enfermedades neuromusculares y rehabilitación. Se realizó una actualización de la bibliografía sobre EPIT, con especial atención en las publicaciones relevantes de los últimos cuatro años. Los términos finales del documento fueron consensuados por todo el grupo de trabajo. Cada participante proporcionó su declaración de conflicto de intereses. El resultado es una actualización del último Consenso Argentino para la EP, con particular enfoque en su forma de comienzo tardío. Tratándose de una afección infrecuente, en la que los datos disponibles son limitados, las presentes recomendaciones deben ser consideradas como opinión de expertos. Pompe’s disease (PD) is an infrequent metabolic autosomic recessive disorder produced by the lack or deficiency of the acid alpha-glucosidase lysosomal enzyme in tissues of involved individuals. Delayed-onset PD is considered whenever symptoms onset start after one year of age. We present an update of the recommendations for the management of delayed-onset PD, taking as reference the guidelines from the Argentine Consensus for diagnosis, treatment and follow-up of PD published in 2013. The present consensus gathered several experts in PD in the areas of internal medicine, laboratory diagnosis, neuropathology, pulmonology, nutrition, neurology, metabolic and neuromuscular disorders as well as rehabilitation to perform an update of the literature of delayed-onset PD, with special attention on relevant information published within the last 4 years. The entire working group approved the final version of the consensus. Each participant provided a declaration of conflict of interest. As a result, it is an update of the previous Argentine PD Consensus with focus on the delayed-onset presentation of the disease. Being such infrequent disorder, available data were rather limited and thus, the recommendations represent expert opinions.

Published
2018

16. Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study

Author

Murai, Hiroyuki, primary, Uzawa, Akiyuki, additional, Suzuki, Yasushi, additional, Imai, Tomihiro, additional, Shiraishi, Hirokazu, additional, Suzuki, Hidekazu, additional, Okumura, Meinoshin, additional, O’Brien, Fanny, additional, Wang, Jing-Jing, additional, Fujita, Kenji P., additional, Utsugisawa, Kimiaki, additional, Mazia, Claudio Gabriel, additional, Wilken, Miguel, additional, Barroso, Fabio, additional, Saba, Juliet, additional, Rugiero, Marcelo, additional, Bettini, Mariela, additional, Chaves, Marcelo, additional, Vidal, Gonzalo, additional, Garcia, Alejandra Dalila, additional, De Bleecker, Jan, additional, Van den Abeele, Guy, additional, de Koning, Kathy, additional, De Mey, Katrien, additional, Mercelis, Rudy, additional, Mahieu, Délphine, additional, Wagemaekers, Linda, additional, Van Damme, Philip, additional, Depreitere, Annelies, additional, Schotte, Caroline, additional, Smetcoren, Charlotte, additional, Stevens, Olivier, additional, Van Daele, Sien, additional, Vandenbussche, Nicolas, additional, Vanhee, Annelies, additional, Verjans, Sarah, additional, Vynckier, Jan, additional, D'Hondt, Ann, additional, Tilkin, Petra, additional, Alves de Siqueira Carvalho, Alzira, additional, Dias Brockhausen, Igor, additional, Feder, David, additional, Ambrosio, Daniel, additional, César, Pamela, additional, Melo, Ana Paula, additional, Martins Ribeiro, Renata, additional, Rocha, Rosana, additional, Bezerra Rosa, Bruno, additional, Veiga, Thabata, additional, da Silva, Luiz Augusto, additional, Santos Engel, Murilo, additional, Gonçalves Geraldo, Jordana, additional, Ananias Morita, Maria da Penha, additional, Nogueira Coelho, Erica, additional, Paiva, Gabriel, additional, Pozo, Marina, additional, Prando, Natalia, additional, Martineli Torres, Debora Dada, additional, Butinhao, Cristiani Fernanda, additional, Duran, Gustavo, additional, Gomes da Silva, Tamires Cristina, additional, Otavio Maia Gonçalves, Luiz, additional, Pazetto, Lucas Eduardo, additional, Fialho, Tomás Augusto Suriane, additional, Renata Cubas Volpe, Luciana, additional, Souza Duca, Luciana, additional, Souza Bulle Oliveira, Acary, additional, Amaral Andrade, Ana Carolina, additional, Annes, Marcelo, additional, Duarte Silva, Liene, additional, Cavalcante Lino, Valeria, additional, Pinto, Wladimir, additional, Assis, Natália, additional, Carrara, Fernanda, additional, Miranda, Carolina, additional, Souza, Iandra, additional, Fernandes, Patricia, additional, Siddiqi, Zaeem, additional, Phan, Cecile, additional, Narayan, Jeffrey, additional, Blackmore, Derrick, additional, Mallon, Ashley, additional, Roderus, Rikki, additional, Watt, Elizabeth, additional, Junkerova, Jana, additional, Kurkova, Barbora, additional, Reguliova, Katarina, additional, Zapletalova, Olga, additional, Pitha, Jiri, additional, Novakova, Iveta, additional, Tyblova, Michaela, additional, Jurajdova, Ivana, additional, Wolfova, Marcela, additional, Andersen, Henning, additional, Harbo, Thomas, additional, Vinge, Lotte, additional, Krogh, Susanne, additional, Mogensen, Anita, additional, Vissing, John, additional, Højgaard, Joan, additional, Witting, Nanna, additional, Mette Ostergaard Autzen, Anne, additional, Pedersen, Jane, additional, Eralinna, Juha-Pekka, additional, Laaksonen, Mikko, additional, Oksaranta, Olli, additional, Harrison, Tuula, additional, Eriksson, Jaana, additional, Rozsa, Csilla, additional, Horvath, Melinda, additional, Lovas, Gabor, additional, Matolcsi, Judit, additional, Szabo, Gyorgyi, additional, Jakab, Gedeonne, additional, Szabadosne, Brigitta, additional, Antonini, Giovanni, additional, Di Pasquale, Antonella, additional, Garibaldi, Matteo, additional, Morino, Stefania, additional, Troili, Fernanda, additional, Fionda, Laura, additional, Filla, Allessandro, additional, Costabile, Teresa, additional, Marano, Enrico, additional, Saccà, Francesco, additional, Fasanaro, Angiola, additional, Marsili, Angela, additional, Puorro, Giorgia, additional, Mantegazza, Renato, additional, Antozzi, Carlo, additional, Bonanno, Silvia, additional, Camera, Giorgia, additional, Locatelli, Alberta, additional, Maggi, Lorenzo, additional, Pasanisi, Maria, additional, Campanella, Angela, additional, Evoli, Amelia, additional, Alboini, Paolo Emilio, additional, D'Amato, Valentina, additional, Iorio, Raffaele, additional, Kanai, Tetsuya, additional, Kawaguchi, Naoki, additional, Mori, Masahiro, additional, Kaneko, Yoko, additional, Kanzaki, Akiko, additional, Kobayashi, Eri, additional, Masaki, Katsuhisa, additional, Matsuse, Dai, additional, Matsushita, Takuya, additional, Uehara, Taira, additional, Shimpo, Misa, additional, Jingu, Maki, additional, Kikutake, Keiko, additional, Nakamura, Yumiko, additional, Sano, Yoshiko, additional, Nagane, Yuriko, additional, Kamegamori, Ikuko, additional, Tsuda, Tomoko, additional, Fujii, Yuko, additional, Futono, Kazumi, additional, Ozawa, Yukiko, additional, Mizugami, Aya, additional, Saito, Yuka, additional, Morikawa, Miyuki, additional, Samukawa, Makoto, additional, Kamakura, Sachiko, additional, Miyawaki, Eriko, additional, Mitazaki, Teiichiro, additional, Motomura, Masakatsu, additional, Mukaino, Akihiro, additional, Yoshimura, Shunsuke, additional, Asada, Shizuka, additional, Yoshida, Seiko, additional, Amamoto, Shoko, additional, Kobashikawa, Tomomi, additional, Koga, Megumi, additional, Maeda, Yasuko, additional, Takada, Kazumi, additional, Takada, Mihoko, additional, Tsurumaru, Masako, additional, Yamashita, Yumi, additional, Akiyama, Tetsuya, additional, Narikawa, Koichi, additional, Tano, Ohito, additional, Tsukita, Kenichi, additional, Kurihara, Rikako, additional, Meguro, Fumie, additional, Fukuda, Yusuke, additional, Sato, Miwako, additional, Funaka, Soichiro, additional, Kawamura, Tomohiro, additional, Makamori, Masayuki, additional, Takahashi, Masanori, additional, Taichi, Namie, additional, Hasuike, Tomoya, additional, Higuchi, Eriko, additional, Kobayashi, Hisako, additional, Osakada, Kaori, additional, Tsuda, Emiko, additional, Shimohama, Shun, additional, Hayashi, Takashi, additional, Hisahara, Shin, additional, Kawamata, Jun, additional, Murahara, Takashi, additional, Saitoh, Masaki, additional, Suzuki, Shuichiro, additional, Yamamoto, Daisuke, additional, Ishiyama, Yoko, additional, Ishiyama, Naoko, additional, Noshiro, Mayuko, additional, Takeyama, Rumi, additional, Uwasa, Kaori, additional, Yasuda, Ikuko, additional, van der Kooi, Anneke, additional, de Visser, Marianne, additional, Gibson, Tamar, additional, Casasnovas, Carlos, additional, Alberti Aguilo, Maria Antonia, additional, Homedes-Pedret, Christian, additional, Julia Palacios, Natalia, additional, Diez Porras, Laura, additional, Velez Santamaria, Valentina, additional, Lazaro, Ana, additional, Diez Tejedor, Exuperio, additional, Gomez Salcedo, Pilar, additional, Fernandez-Fournier, Mireya, additional, Lopez Ruiz, Pedro, additional, Rodriguez de Rivera, Francisco Javier, additional, Sastre, Maria, additional, Gamez, Josep, additional, Sune, Pilar, additional, Salvado, Maria, additional, Gili, Gisela, additional, Mazuela, Gonzalo, additional, Illa, Isabel, additional, Cortes Vicente, Elena, additional, Diaz-Manera, Jordi, additional, Querol Gutierrez, Luis Antonio, additional, Rojas Garcia, Ricardo, additional, Vidal, Nuria, additional, Arribas-Ibar, Elisabet, additional, Piehl, Fredrik, additional, Hietala, Albert, additional, Bjarbo, Lena, additional, Sengun, Ihsan, additional, Meherremova, Arzu, additional, Ozcelik, Pinar, additional, Balkan, Bengu, additional, Tuga, Celal, additional, Ugur, Muzeyyen, additional, Erdem-Ozdamar, Sevim, additional, Bekircan-Kurt, Can Ebru, additional, Acar, Nazire Pinar, additional, Yilmaz, Ezgi, additional, Caliskan, Yagmur, additional, Orsel, Gulsah, additional, Efendi, Husnu, additional, Aydinlik, Seda, additional, Cavus, Hakan, additional, Kutlu, Ayse, additional, Becerikli, Gulsah, additional, Semiz, Cansu, additional, Tun, Ozlem, additional, Terzi, Murat, additional, Dogan, Baki, additional, Onar, Musa Kazim, additional, Sen, Sedat, additional, Kirbas Cavdar, Tugce, additional, Veske, Adife, additional, Norwood, Fiona, additional, Dimitriou, Aikaterini, additional, Gollogly, Jakit, additional, Mahdi-Rogers, Mohamed, additional, Seddigh, Arshira, additional, Sokratous, Giannis, additional, Maier, Gal, additional, Sohail, Faisal, additional, Jacob, Saiju, additional, Sadalage, Girija, additional, Torane, Pravin, additional, Brown, Claire, additional, Shah, Amna, additional, Sathasivam, Sivakumar, additional, Arndt, Heike, additional, Davies, Debbie, additional, Watling, Dave, additional, Amato, Anthony, additional, Cochrane, Thomas, additional, Salajegheh, Mohammed, additional, Roe, Kristen, additional, Amato, Katherine, additional, Toska, Shirli, additional, Wolfe, Gil, additional, Silvestri, Nicholas, additional, Patrick, Kara, additional, Zakalik, Karen, additional, Katz, Jonathan, additional, Miller, Robert, additional, Engel, Marguerite, additional, Forshew, Dallas, additional, Bravver, Elena, additional, Brooks, Benjamin, additional, Plevka, Sarah, additional, Burdette, Maryanne, additional, Cunningham, Scott, additional, Sanjak, Mohammad, additional, Kramer, Megan, additional, Nemeth, Joanne, additional, Schommer, Clara, additional, Tierney, Scott, additional, Juel, Vern, additional, Guptill, Jeffrey, additional, Hobson-Webb, Lisa, additional, Massey, Janice, additional, Beck, Kate, additional, Carnes, Donna, additional, Loor, John, additional, Anderson, Amanda, additional, Pascuzzi, Robert, additional, Bodkin, Cynthia, additional, Kincaid, John, additional, Snook, Riley, additional, Guingrich, Sandra, additional, Micheels, Angela, additional, Chaudhry, Vinay, additional, Corse, Andrea, additional, Mosmiller, Betsy, additional, Kelley, Andrea, additional, Ho, Doreen, additional, Srinivasan, Jayashri, additional, Vytopil, Michal, additional, Jara, Jordan, additional, Ventura, Nicholas, additional, Scala, Stephanie, additional, Carter, Cynthia, additional, Donahue, Craig, additional, Herbert, Carol, additional, Weiner, Elaine, additional, Alam, Sharmeen, additional, McKinnon, Jonathan, additional, Haar, Laura, additional, McKinnon, Naya, additional, Alcon, Karan, additional, McKenna, Kaitlyn, additional, Sattar, Nadia, additional, Daniels, Kevin, additional, Jeffery, Dennis, additional, Freimer, Miriam, additional, Hoyle, Joseph Chad, additional, Kissel, John, additional, Agriesti, Julie, additional, Chelnick, Sharon, additional, Mezache, Louisa, additional, Pineda, Colleen, additional, Muharrem, Filiz, additional, Karam, Chafic, additional, Khoury, Julie, additional, Marburger, Tessa, additional, Kaur, Harpreet, additional, Dimitrova, Diana, additional, Gilchrist, James, additional, Agrawal, Brajesh, additional, Elsayed, Mona, additional, Kohlrus, Stephanie, additional, Andoin, Angela, additional, Darnell, Taylor, additional, Golden, Laura, additional, Lokaitis, Barbara, additional, Seelback, Jenna, additional, Muppidi, Srikanth, additional, Goyal, Neelam, additional, Sakamuri, Sarada, additional, So, Yuen T., additional, Paulose, Shirley, additional, Pol, Sabrina, additional, Welsh, Lesly, additional, Bhavaraju-Sanka, Ratna, additional, Tobon Gonzales, Alejandro, additional, Dishman, Lorraine, additional, Jones, Floyd, additional, Gonzalez, Anna, additional, Padilla, Patricia, additional, Saklad, Amy, additional, Silva, Marcela, additional, Kazamel, Mohamed, additional, Alsharabati, Mohammad, additional, Lu, Liang, additional, Nozaki, Kenkichi, additional, Mumfrey-Thomas, Sandi, additional, Woodall, Amy, additional, Mozaffar, Tahseen, additional, Cash, Tiyonnoh, additional, Goyal, Namita, additional, Roy, Gulmohor, additional, Mathew, Veena, additional, Maqsood, Fatima, additional, Minton, Brian, additional, Jones, H. James, additional, Rosenfeld, Jeffrey, additional, Garcia, Rebekah, additional, Echevarria, Laura, additional, Garcia, Sonia, additional, Pulley, Michael, additional, Aranke, Shachie, additional, Berger, Alan Ross, additional, Shah, Jaimin, additional, Shabbir, Yasmeen, additional, Smith, Lisa, additional, Varghese, Mary, additional, Gutmann, Laurie, additional, Gutmann, Ludwig, additional, Jerath, Nivedita, additional, Nance, Christopher, additional, Swenson, Andrea, additional, Olalde, Heena, additional, Kressin, Nicole, additional, Sieren, Jeri, additional, Barohn, Richard, additional, Dimachkie, Mazen, additional, Glenn, Melanie, additional, McVey, April, additional, Pasnoor, Mamatha, additional, Statland, Jeffery, additional, Wang, Yunxia, additional, Liu, Tina, additional, Emmons, Kelley, additional, Jenci, Nicole, additional, Locheke, Jerry, additional, Fondaw, Alex, additional, Johns, Kathryn, additional, Rico, Gabrielle, additional, Walsh, Maureen, additional, Herbelin, Laura, additional, Hafer-Macko, Charlene, additional, Kwan, Justin, additional, Zilliox, Lindsay, additional, Callison, Karen, additional, Young, Valerie, additional, DiSanzo, Beth, additional, Naunton, Kerry, additional, Benatar, Michael, additional, Bilsker, Martin, additional, Sharma, Khema, additional, Cooley, Anne, additional, Reyes, Eliana, additional, Michon, Sara-Claude, additional, Sheldon, Danielle, additional, Steele, Julie, additional, Howard Jr, James, additional, Chopra, Manisha, additional, Traub, Rebecca, additional, Vu, Tuan, additional, Katzin, Lara, additional, McClain, Terry, additional, Harvey, Brittany, additional, Hart, Adam, additional, Huynh, Kristin, additional, Beydoun, Said, additional, Chilingaryan, Amaiak, additional, Doan, Victor, additional, Droker, Brian, additional, Gong, Hui, additional, Karimi, Sanaz, additional, Lin, Frank, additional, Pokala, Krishna, additional, Shah, Akshay, additional, Tran, Anh, additional, Akhter, Salma, additional, Malekniazi, Ali, additional, Tandan, Rup, additional, Hehir, Michael, additional, Waheed, Waqar, additional, Lucy, Shannon, additional, Weiss, Michael, additional, Distad, Jane, additional, Strom, Susan, additional, Downing, Sharon, additional, Kim, Bryan, additional, Nowak, Richard, additional, Dicapua, Daniel, additional, Keung, Benison, additional, Kumar, Aditya, additional, Patwa, Huned, additional, Robeson, Kimberly, additional, Yang, Irene, additional, Nye, Joan, additional, and Vu, Hong, additional

Published
2019
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17. Eculizumab improves fatigue in refractory generalized myasthenia gravis

Author

Andersen, Henning, Mantegazza, Renato, Wang, Jing Jing, O’Brien, Fanny, Patra, Kaushik, Howard, James F., Mazia, Claudio Gabriel, Wilken, Miguel, Barroso, Fabio, Vissing, John, Højgaard, Joan, Witting, Nanna, Autzen, Anne Ostergaard, Pedersen, Jane, Yang, Irene, Nye, Joan, Vu, Hong, Andersen, Henning, Mantegazza, Renato, Wang, Jing Jing, O’Brien, Fanny, Patra, Kaushik, Howard, James F., Mazia, Claudio Gabriel, Wilken, Miguel, Barroso, Fabio, Vissing, John, Højgaard, Joan, Witting, Nanna, Autzen, Anne Ostergaard, Pedersen, Jane, Yang, Irene, Nye, Joan, and Vu, Hong

Abstract

Purpose: To evaluate the effect of eculizumab on perceived fatigue in patients with anti-acetylcholine receptor antibody-positive, refractory, generalized myasthenia gravis (MG) using the Quality of Life in Neurological Disorders (Neuro-QOL) Fatigue subscale, and to evaluate correlations between improvements in Neuro-QOL Fatigue and other clinical endpoints. Methods: Neuro-QOL Fatigue, MG Activities of Daily Living (MG-ADL), Quantitative MG (QMG), and the 15-item MG Quality of Life (MG-QOL15) scales were administered during the phase 3, randomized, placebo-controlled REGAIN study (eculizumab, n = 62; placebo, n = 63) and subsequent open-label extension (OLE). Data were analyzed using repeated-measures models. Correlations between changes in Neuro-QOL Fatigue and in MG-ADL, QMG, and MG-QOL15 scores were determined at REGAIN week 26. Results: At REGAIN week 26, eculizumab-treated patients showed significantly greater improvements in Neuro-QOL Fatigue scores than placebo-treated patients (consistent with improvements in MG-ADL, QMG, and MG-QOL15 scores previously reported in REGAIN). Improvements with eculizumab were sustained through OLE week 52. Correlations between Neuro-QOL Fatigue and MG-QOL15, MG-ADL, and QMG scores were strong for eculizumab-treated patients at REGAIN week 26, and strong, moderate, and weak, respectively, for placebo-treated patients. Conclusions: Compared with placebo, eculizumab was associated with improvements in perceived fatigue that strongly correlated with improvements in MG-specific outcome measures. Trial ID Registration: NCT01997229, NCT02301624.

Published
2019

18. Consistent improvement with eculizumab across muscle groups in myasthenia gravis.

Author

Mantegazza, Renato, O'Brien, Fanny L., Yountz, Marcus, Howard, James F., Gabriel Mazia, Claudio, Wilken, Miguel, Barroso, Fabio, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Chaves, Marcelo, Vidal, Gonzalo, Dalila Garcia, Alejandra, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, Mercelis, Rudy, Mahieu, Délphine, and Wagemaekers, Linda

Subjects
MYASTHENIA gravis, RESPIRATORY muscles, COMPLEMENT inhibition, MUSCLE strength, ACTIVITIES of daily living, MUSCLES
Abstract

Objective: To assess whether eculizumab, a terminal complement inhibitor, improves patient‐ and physician‐reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively) in patients with refractory anti‐acetylcholine receptor antibody‐positive generalized myasthenia gravis across four domains, representing ocular, bulbar, respiratory, and limb/gross motor muscle groups. Methods: Patients with refractory anti‐acetylcholine receptor antibody‐positive generalized myasthenia gravis were randomized 1:1 to receive either placebo or eculizumab during the REGAIN study (NCT01997229). Patients who completed REGAIN were eligible to continue into the open‐label extension trial (NCT02301624) for up to 4 years. The four domain scores of each of the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale recorded throughout REGAIN and through 130 weeks of the open‐label extension were analyzed. Results: Of the 125 patients who participated in REGAIN, 117 enrolled in the open‐label extension; 61 had received placebo and 56 had received eculizumab during REGAIN. Patients experienced rapid improvements in total scores and all four domain scores of both the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale with eculizumab treatment. These improvements were sustained through 130 weeks of the open‐label extension. Interpretation: Eculizumab treatment elicits rapid and sustained improvements in muscle strength across ocular, bulbar, respiratory, and limb/gross motor muscle groups and in associated daily activities in patients with refractory anti‐acetylcholine receptor antibody‐positive generalized myasthenia gravis. [ABSTRACT FROM AUTHOR]

Published
2020
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20. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study

Author

Howard, James F, primary, Utsugisawa, Kimiaki, additional, Benatar, Michael, additional, Murai, Hiroyuki, additional, Barohn, Richard J, additional, Illa, Isabel, additional, Jacob, Saiju, additional, Vissing, John, additional, Burns, Ted M, additional, Kissel, John T, additional, Muppidi, Srikanth, additional, Nowak, Richard J, additional, O'Brien, Fanny, additional, Wang, Jing-Jing, additional, Mantegazza, Renato, additional, Mazia, Claudio Gabriel, additional, Wilken, Miguel, additional, Ortea, Carolina, additional, Saba, Juliet, additional, Rugiero, Marcelo, additional, Bettini, Mariela, additional, Vidal, Gonzalo, additional, Garcia, Alejandra Dalila, additional, Lamont, Phillipa, additional, Leong, Wai-Kuen, additional, Boterhoven, Heidi, additional, Fyfe, Beverly, additional, Roberts, Leslie, additional, Jasinarachchi, Mahi, additional, Willlems, Natasha, additional, Wanschitz, Julia, additional, Löscher, Wolfgang, additional, De Bleecker, Jan, additional, Van den Abeele, Guy, additional, de Koning, Kathy, additional, De Mey, Katrien, additional, Mercelis, Rudy, additional, Wagemaekers, Linda, additional, Mahieu, Delphine, additional, Van Damme, Philip, additional, Smetcoren, Charlotte, additional, Stevens, Olivier, additional, Verjans, Sarah, additional, D'Hondt, Ann, additional, Tilkin, Petra, additional, Alves de Siqueira Carvalho, Alzira, additional, Hasan, Rosa, additional, Dias Brockhausen, Igor, additional, Feder, David, additional, Ambrosio, Daniel, additional, Melo, Ana Paula, additional, Rocha, Rosana, additional, Rosa, Bruno, additional, Veiga, Thabata, additional, Augusto da Silva, Luiz, additional, Gonçalves Geraldo, Jordana, additional, da Penha Morita Ananias, Maria, additional, Nogueira Coelho, Erica, additional, Paiva, Gabriel, additional, Pozo, Marina, additional, Prando, Natalia, additional, Dada Martineli Torres, Debora, additional, Fernanda Butinhao, Cristiani, additional, Coelho, Erica, additional, Renata Cubas Volpe, Luciana, additional, Duran, Gustavo, additional, Gomes da Silva, Tamires Cristina, additional, Otavio Maia Gonçalves, Luiz, additional, Pazetto, Lucas Eduardo, additional, Souza Duca, Luciana, additional, Suriane Fialho, Tomás Augusto, additional, Gheller Friedrich, Maurício André, additional, Guerreiro, Alexandre, additional, Mohr, Henrique, additional, Pereira Martins, Maurer, additional, da Cruz Pacheco, Daiane, additional, Macagnan, Ana Paula, additional, de Cassia Santos, Aline, additional, Bulle Oliveira, Acary Souza, additional, Amaral de Andrade, Ana Carolina, additional, Annes, Marcelo, additional, Cavalcante Lino, Valeria, additional, Pinto, Wladimir, additional, Miranda, Carolina, additional, Carrara, Fernanda, additional, Souza, Iandra, additional, Genge, Angela, additional, Massie, Rami, additional, Campbell, Natasha, additional, Bril, Vera, additional, Katzberg, Hans, additional, Soltani, Mehran, additional, Ng, Eduardo, additional, Siddiqi, Zaeem, additional, Phan, Celile, additional, Blackmore, Derrick, additional, Vohanka, Stanislav, additional, Bednarik, Josef, additional, Chmelikova, Magda, additional, Cierny, Marek, additional, Toncrova, Stanislava, additional, Junkerova, Jana, additional, Kurkova, Barbora, additional, Reguliova, Katarina, additional, Zapletalova, Olga, additional, Pitha, Jiri, additional, Novakova, Iveta, additional, Tyblova, Michaela, additional, Wolfova, Marcela, additional, Jurajdova, Ivana, additional, Andersen, Henning, additional, Harbo, Thomas, additional, Vinge, Lotte, additional, Mogensen, Anita, additional, Højgaard, Joan, additional, Witting, Nanna, additional, Autzen, Anne Mette, additional, Pedersen, Jane, additional, Färkkilä, Markus, additional, Atula, Sari, additional, Nyrhinen, Anne, additional, Erälinna, Juha-Pekka, additional, Laaksonen, Mikko, additional, Oksaranta, Olli, additional, Eriksson, Jaana, additional, Harrison, Tuula, additional, Desnuelle, Claude, additional, Sacconi, Sabrina, additional, Soriani, Marie-Hélène, additional, Decressac, Sonia, additional, Moutarde, Julie, additional, Lahaut, Pauline, additional, Solé, Guilhem, additional, Le Masson, Gwendal, additional, Wielanek-Bachelet, Anne-Cécile, additional, Gaboreau, Morgane, additional, Moreau, Caroline, additional, Wilson, Amy, additional, Vial, Christophe, additional, Bouhour, Françoise, additional, Gervais-Bernard, Helene, additional, Merle, Hélène, additional, Hourquin, Caroline, additional, Lacour, Arnaud, additional, Outteryck, Olivier, additional, Vermersch, Patrick, additional, Zephir, Hélène, additional, Millois, Edouard, additional, Deneve, Michel, additional, Deruelle, Fabienne, additional, Schoser, Benedikt, additional, Wenninger, Stephan, additional, Stangel, Martin, additional, Alvermann, Sascha, additional, Gingele, Stefan, additional, Skripuletz, Thomas, additional, Suehs, Kurt-Wolfram, additional, Trebst, Corinna, additional, Fricke, Karin, additional, Papagiannopoulos, Sotirios, additional, Bostantzopoulou, Sevasti, additional, Vlaikidis, Nicholas, additional, Zampaki, Martha, additional, Papadopoulou, Nikoletta, additional, Mitsikostas, Dimos-Dimitrios, additional, Kasioti, Eleni, additional, Mitropoulou, Efstathia, additional, Charalambous, Despoina, additional, Rozsa, Csilla, additional, Horvath, Melinda, additional, Lovas, Gabor, additional, Matolcsi, Judit, additional, Szabo, Gyorgyi, additional, Szabadosne, Brigitta, additional, Vecsei, Laszlo, additional, Dezsi, Livia, additional, Varga, Edina, additional, Konyane, Monika, additional, Gross, Bella, additional, Azrilin, Olga, additional, Greenbereg, Nelly, additional, Bali Kuperman, Hila, additional, Antonini, Giovanni, additional, Garibaldi, Matteo, additional, Morino, Stefania, additional, Troili, Fernanda, additional, Di Pasquale, Antonella, additional, Filla, Alessandro, additional, Costabile, Teresa, additional, Marano, Enrico, additional, Sacca, Francesco, additional, Marsil, Angela, additional, Puorro, Giorgia, additional, Maestri Tassoni, Michelangelo, additional, De Rosa, Anna, additional, Bonanno, Silvia, additional, Antozzi, Carlo, additional, Maggi, Lorenzo, additional, Campanella, Angela, additional, Angelini, Corrado, additional, Cudia, Paola, additional, Pegoraro, Valentina, additional, Pinzan, Elena, additional, Bevilacqua, Francesca, additional, Orrico, Daniele, additional, Bonifati, Domenico Marco, additional, Evoli, Amelia, additional, Alboini, Paolo Emilio, additional, D'Amato, Valentina, additional, Iorio, Raffaele, additional, Inghilleri, Maurizio, additional, Fionda, Laura, additional, Frasca, Vittorio, additional, Giacomelli, Elena, additional, Gori, Maria, additional, Lopergolo, Diego, additional, Onesti, Emanuela, additional, Gabriele, Maria, additional, Patti, Francesco, additional, Salvatore Caramma, Andrea, additional, Messina, Silvia, additional, Reggio, Ester, additional, Caserta, Cinzia, additional, Uzawa, Akiyuki, additional, Kanai, Tetsuya, additional, Mori, Masahiro, additional, Kaneko, Yoko, additional, Kanzaki, Akiko, additional, Kobayashi, Eri, additional, Masaki, Katsuhisa, additional, Matsuse, Dai, additional, Matsushita, Takuya, additional, Uehara, Taira, additional, Shimpo, Misa, additional, Jingu, Maki, additional, Kikutake, Keiko, additional, Nakamura, Yumiko, additional, Sano, Yoshiko, additional, Nagane, Yuriko, additional, Kamegamori, Ikuko, additional, Fujii, Yuko, additional, Futono, Kazumi, additional, Tsuda, Tomoko, additional, Saito, Yuka, additional, Suzuki, Hidekazu, additional, Morikawa, Miyuki, additional, Samukawa, Makoto, additional, Kamakura, Sachiko, additional, Shiraishi, Hirokazu, additional, Mitazaki, Teiichiro, additional, Motomura, Masakatsu, additional, Mukaino, Akihiro, additional, Yoshimura, Shunsuke, additional, Asada, Shizuka, additional, Kobashikawa, Tomomi, additional, Koga, Megumi, additional, Maeda, Yasuko, additional, Takada, Kazumi, additional, Takada, Mihoko Takada, additional, Yamashita, Yumi, additional, Yoshida, Seiko, additional, Suzuki, Yasushi, additional, Akiyama, Tetsuya, additional, Narikawa, Koichi, additional, Tsukita, Kenichi, additional, Meguro, Fumie, additional, Fukuda, Yusuke, additional, Sato, Miwako, additional, Matsuo, Hidenori, additional, Fukudome, Takayasu, additional, Gondo, Yuichiro, additional, Maeda, Yasuhiro, additional, Nagaishi, Akiko, additional, Nakane, Shunya, additional, Okubo, Yoshinori, additional, Okumura, Meinoshin, additional, Funaka, Soichiro, additional, Kawamura, Tomohiro, additional, Makamori, Masayuki, additional, Takahashi, Masanori, additional, Hasuike, Tomoya, additional, Higuchi, Eriko, additional, Kobayashi, Hisako, additional, Osakada, Kaori, additional, Taichi, Namie, additional, Tsuda, Emiko, additional, Hayashi, Takashi, additional, Hisahara, Shin, additional, Imai, Tomihiro, additional, Kawamata, Jun, additional, Murahara, Takashi, additional, Saitoh, Masaki, additional, Shimohama, Shun, additional, Suzuki, Shuichiro, additional, Yamamoto, Daisuke, additional, Konno, Shingo, additional, Imamura, Tomomi, additional, Inoue, Masashi, additional, Murata, Mayumi, additional, Nakazora, Hiroshi, additional, Nakayama, Ritsu, additional, Ikeda, Yasuko, additional, Ogawa, Miki, additional, Shirane, Maoko, additional, Kanda, Takashi, additional, Kawai, Motoharu, additional, Koga, Michiaki, additional, Ogasawara, Junichi, additional, Omoto, Masatoshi, additional, Sano, Yasuteru, additional, Arima, Hideki, additional, Fukui, Sachie, additional, Shimose, Shigemi, additional, Shinozaki, Hirokazu, additional, Watanabe, Masanori, additional, Yoshikawa, Chieko, additional, van der Kooi, Anneke, additional, de Visser, Marianne, additional, Gibson, Tamar, additional, Maessen, Jos, additional, de Baets, Marc, additional, Faber, Catherine, additional, Keijzers, Maria Johanna, additional, Miesen, Monique, additional, Kostera-Pruszczyk, Anna, additional, Kaminska, Anna, additional, Kim, Byung-Jo, additional, Lee, Chang Nyoung, additional, Koo, Yong Seo, additional, Seok, Hung Youl, additional, Kang, Hoo Nam, additional, Ra, HyeJin, additional, Kim, Byoung Joon, additional, Cho, Eun Bin, additional, Lee, HyeLim, additional, Min, Ju-Hong, additional, Seok, Jinmyoung, additional, Koh, Da Yoon, additional, Kwon, JuYoung, additional, Lee, JiEun, additional, Park, SangAe, additional, Hong, Yoon-Ho, additional, Lim, Jae-Sung, additional, Kim, MiRi, additional, Kim, Seung Min, additional, Kim, Yool-hee, additional, Lee, Hyung Seok, additional, Shin, Ha Young, additional, Hwang, Eun Bi, additional, Shin, MiJu, additional, Sazonov, Denis, additional, Yarmoschuk, Asya, additional, Babenko, Larisa, additional, Malkova, Nadezhda, additional, Melnikova, Anna, additional, Korobko, Denis, additional, Kosykh, Evgeniya, additional, Pokhabov, Dmitry, additional, Nesterova, Yulia, additional, Abramov, Vladislav, additional, Balyazin, Victor, additional, Casasnovas Pons, Carlos, additional, Alberti Aguilo, Maria, additional, Homedes-Pedret, Christian, additional, Palacios, Natalia Julia, additional, Lazaro, Ana, additional, Diez Tejedor, Exuperio, additional, Fernandez-Fournier, Mireya, additional, Lopez Ruiz, Pedro, additional, Rodriguez de Rivera, Francisco Javier, additional, Salvado Figueras, Maria, additional, Gamez, Josep, additional, Salvado, Maria, additional, Cortes Vicente, Elena, additional, Diaz-Manera, Jordi, additional, Querol Gutierrez, Luis, additional, Rojas Garcia, Ricardo, additional, Vidal, Nuria, additional, Arribas-Ibar, Elisabet, additional, Piehl, Fredrik, additional, Hietala, Albert, additional, Bjarbo, Lena, additional, Lindberg, Christopher, additional, Jons, Daniel, additional, Andersson, Blanka, additional, Sengun, Ihsan, additional, Ozcelik, Pinar, additional, Tuga, Celal, additional, Ugur, Muzeyyen, additional, Boz, Cavit, additional, Altiparmak, Didem, additional, Gazioglu, Sibel, additional, Ozen Aydin, Cigdem, additional, Erdem-Ozdamar, Sevim, additional, Bekircan-Kurt, Can Ebru, additional, Yilmaz, Ezgi, additional, Acar, Nazire Pinar, additional, Caliskan, Yagmur, additional, Efendi, Husnu, additional, Aydinlik, Seda, additional, Cavus, Hakan, additional, Semiz, Cansu, additional, Tun, Ozlem, additional, Terzi, Murat, additional, Dogan, Baki, additional, Onar, Musa Kazim, additional, Sen, Sedat, additional, Cavdar, Tugce Kirbas, additional, Norwood, Fiona, additional, Dimitriou, Aikaterini, additional, Gollogly, Jakit, additional, Mahdi-Rogers, Mohamed, additional, Seddigh, Arshira, additional, Maier, Gal, additional, Sohail, Faisal, additional, Sathasivam, Sivakumar, additional, Arndt, Heike, additional, Davies, Debbie, additional, Watling, Dave, additional, Rivner, Michael, additional, Hartmann, J. Edward, additional, Quarles, Brandy, additional, Smalley, Nicole, additional, Amato, Anthony, additional, Cochrane, Thomas, additional, Salajegheh, Mohammed, additional, Roe, Kristen, additional, Amato, Katherine, additional, Toska, Shirli, additional, Wolfe, Gil, additional, Silvestri, Nicholas, additional, Patrick, Kara, additional, Zakalik, Karen, additional, Katz, Jonathan, additional, Miller, Robert, additional, Engel, Marguerite, additional, Bravver, Elena, additional, Brooks, Benjamin, additional, Plevka, Sarah, additional, Burdette, Maryanne, additional, Sanjak, Mohammad, additional, Kramer, Megan, additional, Nemeth, Joanne, additional, Schommer, Clara, additional, Juel, Vern, additional, Guptill, Jeffrey, additional, Hobson-Webb, Lisa, additional, Beck, Kate, additional, Carnes, Donna, additional, Loor, John, additional, Anderson, Amanda, additional, Lange, Dale, additional, Agopian, Eliz, additional, Goldstein, Jonathan, additional, Manning, Erin, additional, Kaplan, Lindsay, additional, Holzberg, Shara, additional, Kassebaum, Nicole, additional, Pascuzzi, Robert, additional, Bodkin, Cynthia, additional, Kincaid, John, additional, Snook, Riley, additional, Guinrich, Sandra, additional, Micheels, Angela, additional, Chaudhry, Vinay, additional, Corse, Andrea, additional, Mosmiller, Betsy, additional, Ho, Doreen, additional, Srinivasan, Jayashri, additional, Vytopil, Michael, additional, Ventura, Nicholas, additional, Scala, Stephanie, additional, Carter, Cynthia, additional, Donahue, Craig, additional, Herbert, Carol, additional, Weiner, Elaine, additional, McKinnon, Jonathan, additional, Haar, Laura, additional, McKinnon, Naya, additional, Alcon, Karan, additional, Daniels, Kevin, additional, Sattar, Nadia, additional, Jeffery, Dennis, additional, McKenna, Kaitlyn, additional, Guidon, Amanda, additional, David, William, additional, Dheel, Christina, additional, Levine-Weinberg, Mark, additional, Nigro, Catherine, additional, Simpson, Ericka, additional, Appel, Stanley H, additional, Lai, Eugene, additional, Lay, Jr, Luis, additional, Pleitez, Milvia, additional, Halton, Sharon, additional, Faigle, Casey, additional, Thompson, Lisa, additional, Sivak, Mark, additional, Shin, Susan, additional, Bratton, Joan, additional, Jacobs, Daniel, additional, Brown, Gavin, additional, Bandukwala, Ibrez, additional, Brown, Morris, additional, Kane, Jennifer, additional, Blount, Ira, additional, Freimer, Miriam, additional, Hoyle, J. Chad, additional, Agriesti, Julie, additional, Khoury, Julie, additional, Marburger, Tessa, additional, Kaur, Harpreet, additional, Dimitrova, Diana, additional, Mellion, Michelle, additional, Sachs, George, additional, Crabtree, Brigid, additional, Keo, Roseann, additional, Perez, Ele Kim, additional, Taber, Sandra, additional, Gilchrist, James, additional, Andoin, Angela, additional, Darnell, Taylor, additional, Goyal, Neelam, additional, Sakamuri, Sarada, additional, So, Yuen T, additional, Welsh, Lesly Welsh, additional, Bhavaraju-Sanka, Ratna, additional, Tobon Gonzalez, Alejandro, additional, Jones, Floyd, additional, Saklad, Amy, additional, Nations, Sharon, additional, Trivedi, Jaya, additional, Hopkins, Steve, additional, Kazamel, Mohamed, additional, Alsharabati, Mohammad, additional, Lu, Liang, additional, Mumfrey-Thomas, Sandi, additional, Woodall, Amy, additional, Richman, David, additional, Butters, Janelle, additional, Lindsay, Molly, additional, Mozaffar, Tahseen, additional, Cash, Tiyonnoh, additional, Goyal, Namita, additional, Roy, Gulmohor, additional, Mathew, Veena, additional, Maqsood, Fatima, additional, Minton, Brian, additional, Jones, H. James, additional, Rosenfeld, Jeffrey, additional, Garcia, Rebekah, additional, Garcia, Sonia, additional, Echevarria, Laura, additional, Pulley, Michael, additional, Aranke, Shachie, additional, Berger, Alan Ross, additional, Shah, Jaimin, additional, Shabbir, Yasmeen, additional, Smith, Lisa, additional, Varghese, Mary, additional, Gutmann, Laurie, additional, Gutmann, Ludwig, additional, Swenson, Andrea, additional, Olalde, Heena, additional, Hafer-Macko, Charlene, additional, Kwan, Justin, additional, Zilliox, Lindsay, additional, Callison, Karen, additional, DiSanzo, Beth, additional, Naunton, Kerry, additional, Bilsker, Martin, additional, Sharma, Khema, additional, Reyes, Eliana, additional, Cooley, Anne, additional, Michon, Sara-Claude, additional, Steele, Julie, additional, Karam, Chafic Karam, additional, Chopra, Manisha, additional, Bird, Shawn, additional, Kaufman, Jacob, additional, Gallatti, Nichole, additional, Vu, Tuan, additional, Katzin, Lara, additional, McClain, Terry, additional, Harvey, Brittany, additional, Hart, Adam, additional, Huynh, Kristin, additional, Beydoun, Said, additional, Chilingaryan, Amaiak, additional, Droker, Brian, additional, Lin, Frank, additional, Shah, Akshay, additional, Tran, Anh, additional, Akhter, Salma, additional, Malekniazi, Ali, additional, Tandan, Rup, additional, Hehir, Michael, additional, Waheed, Waqar, additional, Lucy, Shannon, additional, Weiss, Michael, additional, Distad, Jane, additional, Downing, Sharon, additional, Strom, Susan, additional, Lisak, Robert, additional, Bernitsas, Evanthia, additional, Khan, Omar, additional, Kumar Sriwastava, Shitiz, additional, Tselis, Alexandros, additional, Jia, Kelly, additional, Bertorini, Tulio, additional, Arnold, Thomas, additional, Henderson, Kendrick, additional, Pillai, Rekha, additional, Liu, Ye, additional, Wheeler, Lauren, additional, Hewlett, Jasmine, additional, Vanderhook, Mollie, additional, Dicapua, Daniel, additional, Keung, Benison, additional, Kumar, Aditya, additional, Patwa, Huned, additional, Robeson, Kimberly, additional, Nye, Joan, additional, and Vu, Hong, additional

Published
2017
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21. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study

Author

Howard, James F, Utsugisawa, Kimiaki, Benatar, Michael, Murai, Hiroyuki, Barohn, Richard J, Illa, Isabel, Jacob, Saiju, Vissing, John, Burns, Ted M, Kissel, John T, Muppidi, Srikanth, Nowak, Richard J, O'Brien, Fanny, Wang, Jing-Jing, Mantegazza, Renato, Mazia, Claudio Gabriel, Wilken, Miguel, Ortea, Carolina, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Vidal, Gonzalo, Garcia, Alejandra Dalila, Lamont, Phillipa, Leong, Wai-Kuen, Boterhoven, Heidi, Fyfe, Beverly, Roberts, Leslie, Jasinarachchi, Mahi, Willlems, Natasha, Wanschitz, Julia, Löscher, Wolfgang, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, Mercelis, Rudy, Wagemaekers, Linda, Mahieu, Delphine, Van Damme, Philip, Smetcoren, Charlotte, Stevens, Olivier, Verjans, Sarah, D'Hondt, Ann, Tilkin, Petra, Alves de Siqueira Carvalho, Alzira, Hasan, Rosa, Dias Brockhausen, Igor, Feder, David, Ambrosio, Daniel, Melo, Ana Paula, Rocha, Rosana, Rosa, Bruno, Veiga, Thabata, Augusto da Silva, Luiz, Gonçalves Geraldo, Jordana, da Penha Morita Ananias, Maria, Nogueira Coelho, Erica, Paiva, Gabriel, Pozo, Marina, Prando, Natalia, Dada Martineli Torres, Debora, Fernanda Butinhao, Cristiani, Coelho, Erica, Renata Cubas Volpe, Luciana, Duran, Gustavo, Gomes da Silva, Tamires Cristina, Otavio Maia Gonçalves, Luiz, Pazetto, Lucas Eduardo, Souza Duca, Luciana, Suriane Fialho, Tomás Augusto, Gheller Friedrich, Maurício André, Guerreiro, Alexandre, Mohr, Henrique, Pereira Martins, Maurer, da Cruz Pacheco, Daiane, Macagnan, Ana Paula, de Cassia Santos, Aline, Bulle Oliveira, Acary Souza, Amaral de Andrade, Ana Carolina, Annes, Marcelo, Cavalcante Lino, Valeria, Pinto, Wladimir, Miranda, Carolina, Carrara, Fernanda, Souza, Iandra, Genge, Angela, Massie, Rami, Campbell, Natasha, Bril, Vera, Katzberg, Han, Soltani, Mehran, Ng, Eduardo, Siddiqi, Zaeem, Phan, Celile, Blackmore, Derrick, Vohanka, Stanislav, Bednarik, Josef, Chmelikova, Magda, Cierny, Marek, Toncrova, Stanislava, Junkerova, Jana, Kurkova, Barbora, Reguliova, Katarina, Zapletalova, Olga, Pitha, Jiri, Novakova, Iveta, Tyblova, Michaela, Wolfova, Marcela, Jurajdova, Ivana, Andersen, Henning, Harbo, Thoma, Vinge, Lotte, Mogensen, Anita, Højgaard, Joan, Witting, Nanna, Autzen, Anne Mette, Pedersen, Jane, Färkkilä, Marku, Atula, Sari, Nyrhinen, Anne, Erälinna, Juha-Pekka, Laaksonen, Mikko, Oksaranta, Olli, Eriksson, Jaana, Harrison, Tuula, Desnuelle, Claude, Sacconi, Sabrina, Soriani, Marie-Hélène, Decressac, Sonia, Moutarde, Julie, Lahaut, Pauline, Solé, Guilhem, Le Masson, Gwendal, Wielanek-Bachelet, Anne-Cécile, Gaboreau, Morgane, Moreau, Caroline, Wilson, Amy, Vial, Christophe, Bouhour, Françoise, Gervais-Bernard, Helene, Merle, Hélène, Hourquin, Caroline, Lacour, Arnaud, Outteryck, Olivier, Vermersch, Patrick, Zephir, Hélène, Millois, Edouard, Deneve, Michel, Deruelle, Fabienne, Schoser, Benedikt, Wenninger, Stephan, Stangel, Martin, Alvermann, Sascha, Gingele, Stefan, Skripuletz, Thoma, Suehs, Kurt-Wolfram, Trebst, Corinna, Fricke, Karin, Papagiannopoulos, Sotirio, Bostantzopoulou, Sevasti, Vlaikidis, Nichola, Zampaki, Martha, Papadopoulou, Nikoletta, Mitsikostas, Dimos-Dimitrio, Kasioti, Eleni, Mitropoulou, Efstathia, Charalambous, Despoina, Rozsa, Csilla, Horvath, Melinda, Lovas, Gabor, Matolcsi, Judit, Szabo, Gyorgyi, Szabadosne, Brigitta, Vecsei, Laszlo, Dezsi, Livia, Varga, Edina, Konyane, Monika, Gross, Bella, Azrilin, Olga, Greenbereg, Nelly, Bali Kuperman, Hila, Antonini, Giovanni, Garibaldi, Matteo, Morino, Stefania, Troili, Fernanda, Di Pasquale, Antonella, Filla, Alessandro, Costabile, Teresa, Marano, Enrico, Sacca, Francesco, Marsil, Angela, Puorro, Giorgia, Maestri Tassoni, Michelangelo, De Rosa, Anna, Bonanno, Silvia, Antozzi, Carlo, Maggi, Lorenzo, Campanella, Angela, Angelini, Corrado, Cudia, Paola, Pegoraro, Valentina, Pinzan, Elena, Bevilacqua, Francesca, Orrico, Daniele, Bonifati, Domenico Marco, Evoli Stampanoni-B, Amelia, Alboini, Paolo Emilio, D'Amato, Valentina, Iorio, Raffaele, Inghilleri, Maurizio, Fionda, Laura, Frasca, Vittorio, Giacomelli, Elena, Gori, Maria, Lopergolo, Diego, Onesti, Emanuela, Gabriele, Maria, Patti, Francesco, Salvatore Caramma, Andrea, Messina, Silvia, Reggio, Ester, Caserta, Cinzia, Uzawa, Akiyuki, Kanai, Tetsuya, Mori, Masahiro, Kaneko, Yoko, Kanzaki, Akiko, Kobayashi, Eri, Masaki, Katsuhisa, Matsuse, Dai, Matsushita, Takuya, Uehara, Taira, Shimpo, Misa, Jingu, Maki, Kikutake, Keiko, Nakamura, Yumiko, Sano, Yoshiko, Nagane, Yuriko, Kamegamori, Ikuko, Fujii, Yuko, Futono, Kazumi, Tsuda, Tomoko, Saito, Yuka, Suzuki, Hidekazu, Morikawa, Miyuki, Samukawa, Makoto, Kamakura, Sachiko, Shiraishi, Hirokazu, Mitazaki, Teiichiro, Motomura, Masakatsu, Mukaino, Akihiro, Yoshimura, Shunsuke, Asada, Shizuka, Kobashikawa, Tomomi, Koga, Megumi, Maeda, Yasuko, Takada, Kazumi, Takada, Mihoko Takada, Yamashita, Yumi, Yoshida, Seiko, Suzuki, Yasushi, Akiyama, Tetsuya, Narikawa, Koichi, Tsukita, Kenichi, Meguro, Fumie, Fukuda, Yusuke, Sato, Miwako, Matsuo, Hidenori, Fukudome, Takayasu, Gondo, Yuichiro, Maeda, Yasuhiro, Nagaishi, Akiko, Nakane, Shunya, Okubo, Yoshinori, Okumura, Meinoshin, Funaka, Soichiro, Kawamura, Tomohiro, Makamori, Masayuki, Takahashi, Masanori, Hasuike, Tomoya, Higuchi, Eriko, Kobayashi, Hisako, Osakada, Kaori, Taichi, Namie, Tsuda, Emiko, Hayashi, Takashi, Hisahara, Shin, Imai, Tomihiro, Kawamata, Jun, Murahara, Takashi, Saitoh, Masaki, Shimohama, Shun, Suzuki, Shuichiro, Yamamoto, Daisuke, Konno, Shingo, Imamura, Tomomi, Inoue, Masashi, Murata, Mayumi, Nakazora, Hiroshi, Nakayama, Ritsu, Ikeda, Yasuko, Ogawa, Miki, Shirane, Maoko, Kanda, Takashi, Kawai, Motoharu, Koga, Michiaki, Ogasawara, Junichi, Omoto, Masatoshi, Sano, Yasuteru, Arima, Hideki, Fukui, Sachie, Shimose, Shigemi, Shinozaki, Hirokazu, Watanabe, Masanori, Yoshikawa, Chieko, van der Kooi, Anneke, de Visser, Marianne, Gibson, Tamar, Maessen, Jo, de Baets, Marc, Faber, Catherine, Keijzers, Maria Johanna, Miesen, Monique, Kostera-Pruszczyk, Anna, Kaminska, Anna, Kim, Byung-Jo, Lee, Chang Nyoung, Koo, Yong Seo, Seok, Hung Youl, Kang, Hoo Nam, Ra, Hyejin, Kim, Byoung Joon, Cho, Eun Bin, Lee, Hyelim, Min, Ju-Hong, Seok, Jinmyoung, Koh, Da Yoon, Kwon, Juyoung, Lee, Jieun, Park, Sangae, Hong, Yoon-Ho, Lim, Jae-Sung, Kim, Miri, Kim, Seung Min, Kim, Yool-hee, Lee, Hyung Seok, Shin, Ha Young, Hwang, Eun Bi, Shin, Miju, Sazonov, Deni, Yarmoschuk, Asya, Babenko, Larisa, Malkova, Nadezhda, Melnikova, Anna, Korobko, Deni, Kosykh, Evgeniya, Pokhabov, Dmitry, Nesterova, Yulia, Abramov, Vladislav, Balyazin, Victor, Casasnovas Pons, Carlo, Alberti Aguilo, Maria, Homedes-Pedret, Christian, Palacios, Natalia Julia, Lazaro, Ana, Diez Tejedor, Exuperio, Fernandez-Fournier, Mireya, Lopez Ruiz, Pedro, Rodriguez de Rivera, Francisco Javier, Salvado Figueras, Maria, Gamez, Josep, Salvado, Maria, Cortes Vicente, Elena, Diaz-Manera, Jordi, Querol Gutierrez, Lui, Rojas Garcia, Ricardo, Vidal, Nuria, Arribas-Ibar, Elisabet, Piehl, Fredrik, Hietala, Albert, Bjarbo, Lena, Lindberg, Christopher, Jons, Daniel, Andersson, Blanka, Sengun, Ihsan, Ozcelik, Pinar, Tuga, Celal, Ugur, Muzeyyen, Boz, Cavit, Altiparmak, Didem, Gazioglu, Sibel, Ozen Aydin, Cigdem, Erdem-Ozdamar, Sevim, Bekircan-Kurt, Can Ebru, Yilmaz, Ezgi, Acar, Nazire Pinar, Caliskan, Yagmur, Efendi, Husnu, Aydinlik, Seda, Cavus, Hakan, Semiz, Cansu, Tun, Ozlem, Terzi, Murat, Dogan, Baki, Onar, Musa Kazim, Sen, Sedat, Cavdar, Tugce Kirba, Norwood, Fiona, Dimitriou, Aikaterini, Gollogly, Jakit, Mahdi-Rogers, Mohamed, Seddigh, Arshira, Maier, Gal, Sohail, Faisal, Sathasivam, Sivakumar, Arndt, Heike, Davies, Debbie, Watling, Dave, Rivner, Michael, Hartmann, J. Edward, Quarles, Brandy, Smalley, Nicole, Amato, Anthony, Cochrane, Thoma, Salajegheh, Mohammed, Roe, Kristen, Amato, Katherine, Toska, Shirli, Wolfe, Gil, Silvestri, Nichola, Patrick, Kara, Zakalik, Karen, Katz, Jonathan, Miller, Robert, Engel, Marguerite, Bravver, Elena, Brooks, Benjamin, Plevka, Sarah, Burdette, Maryanne, Sanjak, Mohammad, Kramer, Megan, Nemeth, Joanne, Schommer, Clara, Juel, Vern, Guptill, Jeffrey, Hobson-Webb, Lisa, Beck, Kate, Carnes, Donna, Loor, John, Anderson, Amanda, Lange, Dale, Agopian, Eliz, Goldstein, Jonathan, Manning, Erin, Kaplan, Lindsay, Holzberg, Shara, Kassebaum, Nicole, Pascuzzi, Robert, Bodkin, Cynthia, Kincaid, John, Snook, Riley, Guinrich, Sandra, Micheels, Angela, Chaudhry, Vinay, Corse, Andrea, Mosmiller, Betsy, Ho, Doreen, Srinivasan, Jayashri, Vytopil, Michael, Ventura, Nichola, Scala, Stephanie, Carter, Cynthia, Donahue, Craig, Herbert, Carol, Weiner, Elaine, Mckinnon, Jonathan, Haar, Laura, Mckinnon, Naya, Alcon, Karan, Daniels, Kevin, Sattar, Nadia, Jeffery, Denni, Mckenna, Kaitlyn, Guidon, Amanda, David, William, Dheel, Christina, Levine-Weinberg, Mark, Nigro, Catherine, Simpson, Ericka, Appel, Stanley H, Lai, Eugene, Lay, Lui, Pleitez, Milvia, Halton, Sharon, Faigle, Casey, Thompson, Lisa, Sivak, Mark, Shin, Susan, Bratton, Joan, Jacobs, Daniel, Brown, Gavin, Bandukwala, Ibrez, Brown, Morri, Kane, Jennifer, Blount, Ira, Freimer, Miriam, Hoyle, J. Chad, Agriesti, Julie, Khoury, Julie, Marburger, Tessa, Kaur, Harpreet, Dimitrova, Diana, Mellion, Michelle, Sachs, George, Crabtree, Brigid, Keo, Roseann, Perez, Ele Kim, Taber, Sandra, Gilchrist, Jame, Andoin, Angela, Darnell, Taylor, Goyal, Neelam, Sakamuri, Sarada, So, Yuen T, Welsh, Lesly Welsh, Bhavaraju-Sanka, Ratna, Tobon Gonzalez, Alejandro, Jones, Floyd, Saklad, Amy, Nations, Sharon, Trivedi, Jaya, Hopkins, Steve, Kazamel, Mohamed, Alsharabati, Mohammad, Lu, Liang, Mumfrey-Thomas, Sandi, Woodall, Amy, Richman, David, Butters, Janelle, Lindsay, Molly, Mozaffar, Tahseen, Cash, Tiyonnoh, Goyal, Namita, Roy, Gulmohor, Mathew, Veena, Maqsood, Fatima, Minton, Brian, Jones, H. Jame, Rosenfeld, Jeffrey, Garcia, Rebekah, Garcia, Sonia, Echevarria, Laura, Pulley, Michael, Aranke, Shachie, Berger, Alan Ro, Shah, Jaimin, Shabbir, Yasmeen, Smith, Lisa, Varghese, Mary, Gutmann, Laurie, Gutmann, Ludwig, Swenson, Andrea, Olalde, Heena, Hafer-Macko, Charlene, Kwan, Justin, Zilliox, Lindsay, Callison, Karen, Disanzo, Beth, Naunton, Kerry, Bilsker, Martin, Sharma, Khema, Reyes, Eliana, Cooley, Anne, Michon, Sara-Claude, Steele, Julie, Karam, Chafic Karam, Chopra, Manisha, Bird, Shawn, Kaufman, Jacob, Gallatti, Nichole, Vu, Tuan, Katzin, Lara, Mcclain, Terry, Harvey, Brittany, Hart, Adam, Huynh, Kristin, Beydoun, Said, Chilingaryan, Amaiak, Droker, Brian, Lin, Frank, Shah, Akshay, Tran, Anh, Akhter, Salma, Malekniazi, Ali, Tandan, Rup, Hehir, Michael, Waheed, Waqar, Lucy, Shannon, Weiss, Michael, Distad, Jane, Downing, Sharon, Strom, Susan, Lisak, Robert, Bernitsas, Evanthia, Khan, Omar, Kumar Sriwastava, Shitiz, Tselis, Alexandro, Jia, Kelly, Bertorini, Tulio, Arnold, Thoma, Henderson, Kendrick, Pillai, Rekha, Liu, Ye, Wheeler, Lauren, Hewlett, Jasmine, Vanderhook, Mollie, Dicapua, Daniel, Keung, Benison, Kumar, Aditya, Patwa, Huned, Robeson, Kimberly, Nye, Joan, Vu, Hong, Evoli, Amelia (ORCID:0000-0003-0282-8787), Howard, James F, Utsugisawa, Kimiaki, Benatar, Michael, Murai, Hiroyuki, Barohn, Richard J, Illa, Isabel, Jacob, Saiju, Vissing, John, Burns, Ted M, Kissel, John T, Muppidi, Srikanth, Nowak, Richard J, O'Brien, Fanny, Wang, Jing-Jing, Mantegazza, Renato, Mazia, Claudio Gabriel, Wilken, Miguel, Ortea, Carolina, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Vidal, Gonzalo, Garcia, Alejandra Dalila, Lamont, Phillipa, Leong, Wai-Kuen, Boterhoven, Heidi, Fyfe, Beverly, Roberts, Leslie, Jasinarachchi, Mahi, Willlems, Natasha, Wanschitz, Julia, Löscher, Wolfgang, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, Mercelis, Rudy, Wagemaekers, Linda, Mahieu, Delphine, Van Damme, Philip, Smetcoren, Charlotte, Stevens, Olivier, Verjans, Sarah, D'Hondt, Ann, Tilkin, Petra, Alves de Siqueira Carvalho, Alzira, Hasan, Rosa, Dias Brockhausen, Igor, Feder, David, Ambrosio, Daniel, Melo, Ana Paula, Rocha, Rosana, Rosa, Bruno, Veiga, Thabata, Augusto da Silva, Luiz, Gonçalves Geraldo, Jordana, da Penha Morita Ananias, Maria, Nogueira Coelho, Erica, Paiva, Gabriel, Pozo, Marina, Prando, Natalia, Dada Martineli Torres, Debora, Fernanda Butinhao, Cristiani, Coelho, Erica, Renata Cubas Volpe, Luciana, Duran, Gustavo, Gomes da Silva, Tamires Cristina, Otavio Maia Gonçalves, Luiz, Pazetto, Lucas Eduardo, Souza Duca, Luciana, Suriane Fialho, Tomás Augusto, Gheller Friedrich, Maurício André, Guerreiro, Alexandre, Mohr, Henrique, Pereira Martins, Maurer, da Cruz Pacheco, Daiane, Macagnan, Ana Paula, de Cassia Santos, Aline, Bulle Oliveira, Acary Souza, Amaral de Andrade, Ana Carolina, Annes, Marcelo, Cavalcante Lino, Valeria, Pinto, Wladimir, Miranda, Carolina, Carrara, Fernanda, Souza, Iandra, Genge, Angela, Massie, Rami, Campbell, Natasha, Bril, Vera, Katzberg, Han, Soltani, Mehran, Ng, Eduardo, Siddiqi, Zaeem, Phan, Celile, Blackmore, Derrick, Vohanka, Stanislav, Bednarik, Josef, Chmelikova, Magda, Cierny, Marek, Toncrova, Stanislava, Junkerova, Jana, Kurkova, Barbora, Reguliova, Katarina, Zapletalova, Olga, Pitha, Jiri, Novakova, Iveta, Tyblova, Michaela, Wolfova, Marcela, Jurajdova, Ivana, Andersen, Henning, Harbo, Thoma, Vinge, Lotte, Mogensen, Anita, Højgaard, Joan, Witting, Nanna, Autzen, Anne Mette, Pedersen, Jane, Färkkilä, Marku, Atula, Sari, Nyrhinen, Anne, Erälinna, Juha-Pekka, Laaksonen, Mikko, Oksaranta, Olli, Eriksson, Jaana, Harrison, Tuula, Desnuelle, Claude, Sacconi, Sabrina, Soriani, Marie-Hélène, Decressac, Sonia, Moutarde, Julie, Lahaut, Pauline, Solé, Guilhem, Le Masson, Gwendal, Wielanek-Bachelet, Anne-Cécile, Gaboreau, Morgane, Moreau, Caroline, Wilson, Amy, Vial, Christophe, Bouhour, Françoise, Gervais-Bernard, Helene, Merle, Hélène, Hourquin, Caroline, Lacour, Arnaud, Outteryck, Olivier, Vermersch, Patrick, Zephir, Hélène, Millois, Edouard, Deneve, Michel, Deruelle, Fabienne, Schoser, Benedikt, Wenninger, Stephan, Stangel, Martin, Alvermann, Sascha, Gingele, Stefan, Skripuletz, Thoma, Suehs, Kurt-Wolfram, Trebst, Corinna, Fricke, Karin, Papagiannopoulos, Sotirio, Bostantzopoulou, Sevasti, Vlaikidis, Nichola, Zampaki, Martha, Papadopoulou, Nikoletta, Mitsikostas, Dimos-Dimitrio, Kasioti, Eleni, Mitropoulou, Efstathia, Charalambous, Despoina, Rozsa, Csilla, Horvath, Melinda, Lovas, Gabor, Matolcsi, Judit, Szabo, Gyorgyi, Szabadosne, Brigitta, Vecsei, Laszlo, Dezsi, Livia, Varga, Edina, Konyane, Monika, Gross, Bella, Azrilin, Olga, Greenbereg, Nelly, Bali Kuperman, Hila, Antonini, Giovanni, Garibaldi, Matteo, Morino, Stefania, Troili, Fernanda, Di Pasquale, Antonella, Filla, Alessandro, Costabile, Teresa, Marano, Enrico, Sacca, Francesco, Marsil, Angela, Puorro, Giorgia, Maestri Tassoni, Michelangelo, De Rosa, Anna, Bonanno, Silvia, Antozzi, Carlo, Maggi, Lorenzo, Campanella, Angela, Angelini, Corrado, Cudia, Paola, Pegoraro, Valentina, Pinzan, Elena, Bevilacqua, Francesca, Orrico, Daniele, Bonifati, Domenico Marco, Evoli Stampanoni-B, Amelia, Alboini, Paolo Emilio, D'Amato, Valentina, Iorio, Raffaele, Inghilleri, Maurizio, Fionda, Laura, Frasca, Vittorio, Giacomelli, Elena, Gori, Maria, Lopergolo, Diego, Onesti, Emanuela, Gabriele, Maria, Patti, Francesco, Salvatore Caramma, Andrea, Messina, Silvia, Reggio, Ester, Caserta, Cinzia, Uzawa, Akiyuki, Kanai, Tetsuya, Mori, Masahiro, Kaneko, Yoko, Kanzaki, Akiko, Kobayashi, Eri, Masaki, Katsuhisa, Matsuse, Dai, Matsushita, Takuya, Uehara, Taira, Shimpo, Misa, Jingu, Maki, Kikutake, Keiko, Nakamura, Yumiko, Sano, Yoshiko, Nagane, Yuriko, Kamegamori, Ikuko, Fujii, Yuko, Futono, Kazumi, Tsuda, Tomoko, Saito, Yuka, Suzuki, Hidekazu, Morikawa, Miyuki, Samukawa, Makoto, Kamakura, Sachiko, Shiraishi, Hirokazu, Mitazaki, Teiichiro, Motomura, Masakatsu, Mukaino, Akihiro, Yoshimura, Shunsuke, Asada, Shizuka, Kobashikawa, Tomomi, Koga, Megumi, Maeda, Yasuko, Takada, Kazumi, Takada, Mihoko Takada, Yamashita, Yumi, Yoshida, Seiko, Suzuki, Yasushi, Akiyama, Tetsuya, Narikawa, Koichi, Tsukita, Kenichi, Meguro, Fumie, Fukuda, Yusuke, Sato, Miwako, Matsuo, Hidenori, Fukudome, Takayasu, Gondo, Yuichiro, Maeda, Yasuhiro, Nagaishi, Akiko, Nakane, Shunya, Okubo, Yoshinori, Okumura, Meinoshin, Funaka, Soichiro, Kawamura, Tomohiro, Makamori, Masayuki, Takahashi, Masanori, Hasuike, Tomoya, Higuchi, Eriko, Kobayashi, Hisako, Osakada, Kaori, Taichi, Namie, Tsuda, Emiko, Hayashi, Takashi, Hisahara, Shin, Imai, Tomihiro, Kawamata, Jun, Murahara, Takashi, Saitoh, Masaki, Shimohama, Shun, Suzuki, Shuichiro, Yamamoto, Daisuke, Konno, Shingo, Imamura, Tomomi, Inoue, Masashi, Murata, Mayumi, Nakazora, Hiroshi, Nakayama, Ritsu, Ikeda, Yasuko, Ogawa, Miki, Shirane, Maoko, Kanda, Takashi, Kawai, Motoharu, Koga, Michiaki, Ogasawara, Junichi, Omoto, Masatoshi, Sano, Yasuteru, Arima, Hideki, Fukui, Sachie, Shimose, Shigemi, Shinozaki, Hirokazu, Watanabe, Masanori, Yoshikawa, Chieko, van der Kooi, Anneke, de Visser, Marianne, Gibson, Tamar, Maessen, Jo, de Baets, Marc, Faber, Catherine, Keijzers, Maria Johanna, Miesen, Monique, Kostera-Pruszczyk, Anna, Kaminska, Anna, Kim, Byung-Jo, Lee, Chang Nyoung, Koo, Yong Seo, Seok, Hung Youl, Kang, Hoo Nam, Ra, Hyejin, Kim, Byoung Joon, Cho, Eun Bin, Lee, Hyelim, Min, Ju-Hong, Seok, Jinmyoung, Koh, Da Yoon, Kwon, Juyoung, Lee, Jieun, Park, Sangae, Hong, Yoon-Ho, Lim, Jae-Sung, Kim, Miri, Kim, Seung Min, Kim, Yool-hee, Lee, Hyung Seok, Shin, Ha Young, Hwang, Eun Bi, Shin, Miju, Sazonov, Deni, Yarmoschuk, Asya, Babenko, Larisa, Malkova, Nadezhda, Melnikova, Anna, Korobko, Deni, Kosykh, Evgeniya, Pokhabov, Dmitry, Nesterova, Yulia, Abramov, Vladislav, Balyazin, Victor, Casasnovas Pons, Carlo, Alberti Aguilo, Maria, Homedes-Pedret, Christian, Palacios, Natalia Julia, Lazaro, Ana, Diez Tejedor, Exuperio, Fernandez-Fournier, Mireya, Lopez Ruiz, Pedro, Rodriguez de Rivera, Francisco Javier, Salvado Figueras, Maria, Gamez, Josep, Salvado, Maria, Cortes Vicente, Elena, Diaz-Manera, Jordi, Querol Gutierrez, Lui, Rojas Garcia, Ricardo, Vidal, Nuria, Arribas-Ibar, Elisabet, Piehl, Fredrik, Hietala, Albert, Bjarbo, Lena, Lindberg, Christopher, Jons, Daniel, Andersson, Blanka, Sengun, Ihsan, Ozcelik, Pinar, Tuga, Celal, Ugur, Muzeyyen, Boz, Cavit, Altiparmak, Didem, Gazioglu, Sibel, Ozen Aydin, Cigdem, Erdem-Ozdamar, Sevim, Bekircan-Kurt, Can Ebru, Yilmaz, Ezgi, Acar, Nazire Pinar, Caliskan, Yagmur, Efendi, Husnu, Aydinlik, Seda, Cavus, Hakan, Semiz, Cansu, Tun, Ozlem, Terzi, Murat, Dogan, Baki, Onar, Musa Kazim, Sen, Sedat, Cavdar, Tugce Kirba, Norwood, Fiona, Dimitriou, Aikaterini, Gollogly, Jakit, Mahdi-Rogers, Mohamed, Seddigh, Arshira, Maier, Gal, Sohail, Faisal, Sathasivam, Sivakumar, Arndt, Heike, Davies, Debbie, Watling, Dave, Rivner, Michael, Hartmann, J. Edward, Quarles, Brandy, Smalley, Nicole, Amato, Anthony, Cochrane, Thoma, Salajegheh, Mohammed, Roe, Kristen, Amato, Katherine, Toska, Shirli, Wolfe, Gil, Silvestri, Nichola, Patrick, Kara, Zakalik, Karen, Katz, Jonathan, Miller, Robert, Engel, Marguerite, Bravver, Elena, Brooks, Benjamin, Plevka, Sarah, Burdette, Maryanne, Sanjak, Mohammad, Kramer, Megan, Nemeth, Joanne, Schommer, Clara, Juel, Vern, Guptill, Jeffrey, Hobson-Webb, Lisa, Beck, Kate, Carnes, Donna, Loor, John, Anderson, Amanda, Lange, Dale, Agopian, Eliz, Goldstein, Jonathan, Manning, Erin, Kaplan, Lindsay, Holzberg, Shara, Kassebaum, Nicole, Pascuzzi, Robert, Bodkin, Cynthia, Kincaid, John, Snook, Riley, Guinrich, Sandra, Micheels, Angela, Chaudhry, Vinay, Corse, Andrea, Mosmiller, Betsy, Ho, Doreen, Srinivasan, Jayashri, Vytopil, Michael, Ventura, Nichola, Scala, Stephanie, Carter, Cynthia, Donahue, Craig, Herbert, Carol, Weiner, Elaine, Mckinnon, Jonathan, Haar, Laura, Mckinnon, Naya, Alcon, Karan, Daniels, Kevin, Sattar, Nadia, Jeffery, Denni, Mckenna, Kaitlyn, Guidon, Amanda, David, William, Dheel, Christina, Levine-Weinberg, Mark, Nigro, Catherine, Simpson, Ericka, Appel, Stanley H, Lai, Eugene, Lay, Lui, Pleitez, Milvia, Halton, Sharon, Faigle, Casey, Thompson, Lisa, Sivak, Mark, Shin, Susan, Bratton, Joan, Jacobs, Daniel, Brown, Gavin, Bandukwala, Ibrez, Brown, Morri, Kane, Jennifer, Blount, Ira, Freimer, Miriam, Hoyle, J. Chad, Agriesti, Julie, Khoury, Julie, Marburger, Tessa, Kaur, Harpreet, Dimitrova, Diana, Mellion, Michelle, Sachs, George, Crabtree, Brigid, Keo, Roseann, Perez, Ele Kim, Taber, Sandra, Gilchrist, Jame, Andoin, Angela, Darnell, Taylor, Goyal, Neelam, Sakamuri, Sarada, So, Yuen T, Welsh, Lesly Welsh, Bhavaraju-Sanka, Ratna, Tobon Gonzalez, Alejandro, Jones, Floyd, Saklad, Amy, Nations, Sharon, Trivedi, Jaya, Hopkins, Steve, Kazamel, Mohamed, Alsharabati, Mohammad, Lu, Liang, Mumfrey-Thomas, Sandi, Woodall, Amy, Richman, David, Butters, Janelle, Lindsay, Molly, Mozaffar, Tahseen, Cash, Tiyonnoh, Goyal, Namita, Roy, Gulmohor, Mathew, Veena, Maqsood, Fatima, Minton, Brian, Jones, H. Jame, Rosenfeld, Jeffrey, Garcia, Rebekah, Garcia, Sonia, Echevarria, Laura, Pulley, Michael, Aranke, Shachie, Berger, Alan Ro, Shah, Jaimin, Shabbir, Yasmeen, Smith, Lisa, Varghese, Mary, Gutmann, Laurie, Gutmann, Ludwig, Swenson, Andrea, Olalde, Heena, Hafer-Macko, Charlene, Kwan, Justin, Zilliox, Lindsay, Callison, Karen, Disanzo, Beth, Naunton, Kerry, Bilsker, Martin, Sharma, Khema, Reyes, Eliana, Cooley, Anne, Michon, Sara-Claude, Steele, Julie, Karam, Chafic Karam, Chopra, Manisha, Bird, Shawn, Kaufman, Jacob, Gallatti, Nichole, Vu, Tuan, Katzin, Lara, Mcclain, Terry, Harvey, Brittany, Hart, Adam, Huynh, Kristin, Beydoun, Said, Chilingaryan, Amaiak, Droker, Brian, Lin, Frank, Shah, Akshay, Tran, Anh, Akhter, Salma, Malekniazi, Ali, Tandan, Rup, Hehir, Michael, Waheed, Waqar, Lucy, Shannon, Weiss, Michael, Distad, Jane, Downing, Sharon, Strom, Susan, Lisak, Robert, Bernitsas, Evanthia, Khan, Omar, Kumar Sriwastava, Shitiz, Tselis, Alexandro, Jia, Kelly, Bertorini, Tulio, Arnold, Thoma, Henderson, Kendrick, Pillai, Rekha, Liu, Ye, Wheeler, Lauren, Hewlett, Jasmine, Vanderhook, Mollie, Dicapua, Daniel, Keung, Benison, Kumar, Aditya, Patwa, Huned, Robeson, Kimberly, Nye, Joan, Vu, Hong, and Evoli, Amelia (ORCID:0000-0003-0282-8787)

Abstract

Background Complement is likely to have a role in refractory generalised myasthenia gravis, but no approved therapies specifically target this system. Results from a phase 2 study suggested that eculizumab, a terminal complement inhibitor, produced clinically meaningful improvements in patients with anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis. We further assessed the efficacy and safety of eculizumab in this patient population in a phase 3 trial. Methods We did a phase 3, randomised, double-blind, placebo-controlled, multicentre study (REGAIN) in 76 hospitals and specialised clinics in 17 countries across North America, Latin America, Europe, and Asia. Eligible patients were aged at least 18 years, with a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score of 6 or more, Myasthenia Gravis Foundation of America (MGFA) class II–IV disease, vaccination against Neisseria meningitides, and previous treatment with at least two immunosuppressive therapies or one immunosuppressive therapy and chronic intravenous immunoglobulin or plasma exchange for 12 months without symptom control. Patients with a history of thymoma or thymic neoplasms, thymectomy within 12 months before screening, or use of intravenous immunoglobulin or plasma exchange within 4 weeks before randomisation, or rituximab within 6 months before screening, were excluded. We randomly assigned participants (1:1) to either intravenous eculizumab or intravenous matched placebo for 26 weeks. Dosing for eculizumab was 900 mg on day 1 and at weeks 1, 2, and 3; 1200 mg at week 4; and 1200 mg given every second week thereafter as maintenance dosing. Randomisation was done centrally with an interactive voice or web-response system with patients stratified to one of four groups based on MGFA disease classification. Where possible, patients were maintained on existing myasthenia gravis therapies and rescue medication was allowed at the study physician's discretion. Pat

Published
2017

24. Adverse Outcome of Early Recurrent Ischemic Stroke Secondary to Atrial Fibrillation after Repeated Systemic Thrombolysis

Author

Sposato, Luciano A., Salutto, Valeria, Beratti, Diego E., Monti, Paula, Riccio, Patricia M., and Mazia, Claudio

Subjects
Article Subject, cardiovascular diseases
Abstract

Background. Recurrent ischemic stroke is associated with adverse neurological outcome in patients with atrial fibrillation. There is very scarce information regarding the neurological outcome of atrial fibrillation patients undergoing repeated systemic thrombolysis after early recurrent ischemic stroke. Clinical Case and Discussion. We describe a case of a 76-year-old woman with known paroxysmal atrial fibrillation who was admitted because of an acute right middle cerebral artery ischemic stroke and who underwent repeated systemic thrombolysis within 110 hours. The patient underwent systemic thrombolysis after the first ischemic stroke with almost complete neurological recovery. On the fourth day after treatment, an acute left middle cerebral artery ischemic stroke was diagnosed and she was treated with full-dose intravenous recombinant tissue plasminogen activator. A hemorrhagic transformation of the left middle cerebral artery infarction was noted on follow-up cranial computed tomographic scans. The patient did not recover from the second cerebrovascular event and died 25 days after admission. Conclusion. To the best of our knowledge, this is the second case reporting the adverse neurological outcome of a patient with diagnosis of atrial fibrillation undergoing repeated systemic thrombolysis after early recurrent ischemic stroke. Our report represents a contribution to the scarce available evidence suggesting that repeated systemic thrombolysis for recurrent ischemic stroke should be avoided.

Published
2013
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25. Screening of the intronic expansion in the C9 orf72 gene and the ATXN2 mutation in Argentine patients diagnosed with Amyotrophic Lateral Sclerosis (P2.056)

Author

Tillard, Belén, primary, Cisneros, Elisa, additional, Marchesoni, Cintia, additional, Pardal, Ana, additional, Schottlaender, Lucía, additional, Houlden, Henry, additional, M, Shoai, additional, Fulgenzi, Ernesto, additional, Conti, Eugenia, additional, José, Di Pace, additional, Carnero, Edgar, additional, Perez Akly, Manuel, additional, Nogués, Martín, additional, Uchitel, Osvaldo, additional, de Ambrosi, Bruno, additional, Lautre, Andrea, additional, Barroso, Fabio Adrian, additional, Dubrovsky, Alberto, additional, Pirra, Laura, additional, Jauregui, Agustin, additional, Landriscina, Paula, additional, Rodriguez, Alejandro, additional, Goyeneche, Paula, additional, Mazia, Claudio, additional, Salutto, Valeria Lujan, additional, Nofal, Pedro, additional, Salazar, Zulma, additional, Brand, Patricio, additional, Surace, Ezequiel, additional, and Reisin, Ricardo, additional

Published
2015
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27. Multicenter Epidemiological Study of Amyotrophic Lateral Sclerosis in the City of Buenos Aires (P2.057)

Author

Manuel, Pérez Akly, primary, Albanese, Gustavo, additional, Barroso, Fabio Adrian, additional, Bendersky, Mariana, additional, Bettini, Mariela, additional, Di Egidio, Marianna, additional, Fulgenzi, Ernesto, additional, Fiorotto, Luís, additional, Gargiulo Monachelli, Gisella, additional, Jáuregui, Agustín, additional, Landriscina, Paula, additional, Mazia, Claudio, additional, Melcom, Mario, additional, Reisin, Ricardo, additional, Rey, Roberto, additional, Rodríguez, Gabriel, additional, Rugiero, Marcelo Francisco, additional, Salutto, Valeria Lujan, additional, and Tillard, Belén, additional

Published
2015
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28. Inmunoglobulina endovenosa en enfermedades neuromusculares. Guía para su utilización

Author

Figueredo, Alejandra, Altamirano, Lorena, Amores, Mirtha Graciela, Bertotti, Alicia Cristina, Cueto, Alicia, Díaz Livadiotis, Guillermo, Di Egidio, Mariana, Doumic, Javier, Dubrovsky, Alberto, Fulgenzi, Ernesto, Lautre, Andrea, Losavio, Adriana Silvia, Marchesone, Cintia, Martinez Alvarez, Mariana, Mazia, Claudio Gabriel, Melano, Raúl, Orellano, Anabel, Pagano, Miguel Angel, Pardal, Ana Maria, Pirra, Laura, Politei, Juan, Reisin, Ricardo, Rey, Roberto, Rodriguez, Gabriel, Rugiero, Marcelo, and Yorio, Alberto

Subjects
Medicina Básica, CIENCIAS MÉDICAS Y DE LA SALUD, Fisiología
Abstract

La Inmunoglobulina Endovenosa (IgEV) ha mostrado eficacia en varias enfermedades inmunomediadas del Sistema Nervioso Periférico. Los mecanismos postulados son: inhibir la producción de autoanticuerpos, neutralizarlos y aumentar su catabolismo, inducir bloqueo sobre monocitos y células T, interferir con el Complemento e interactuar con diversas citoquinas. La IgEV es elaborada a partir de la purificación y concentración del plasma de individuos sanos; aplicando diversas metodologías, como tratamiento a pH ácido con trazas de pepsina, cromatografía de intercambio iónico y precipitación con polietilenglicol, para eliminar los polímeros de alto peso molecular. Las entidades en las cuales se evaluó la IgEV más frecuentemente son: Neuropatías inmunomediadas Agudas (Síndrome Guillain-Barré y sus variedades: Síndrome de Miller-Fisher, Neuropatía Axonal Aguda Motora y Neuropatía Axonal Aguda Motora y Sensitiva) y Crónicas (Polineuropatía Inflamatoria Desmielinizante Crónica, Neuropatía Multifocal Motora, Neuropatía Desmielinizante Multifocal Sensitiva y Motora, Neuropatías Asociadas a Paraproteinemia y Neuropatías Atáxicas Crónicas Predominantemente Sensitivas), Enfermedades de la Unión Neuromuscular (Miastenia Gravis y Síndrome de Eaton-Lambert), Miopatías Inflamatorias (Dermatomiositis, Polimiositis y Miositis por Cuerpos de Inclusión) Ganglionopatías Sensitivas y Síndrome de Persona Rígida. La IgEV es fácilmente administrable y generalmente bien tolerada. Los efectos adversos raramente son serios, frecuentemente escalofríos, náuseas, cefalea, mialgias, fatiga y fiebre entre otros, controlables con tratamiento sintomático, y raramente falla renal, infartos miocárdicos, accidentes cerebrovasculares, reacción anafiláctica y meningitis aséptica. La IgEV se contraindica en hipersensibilidad a Inmunoglobulinas y en pacientes con déficit congénito de IgA. La dosis es de 2 g/kg, Clásicamente se distribuye a lo largo de 2-5 días con velocidad de infusión de 40-80 ml/hora. Fil: Figueredo, Alejandra. Sociedad Neurológica Argentina; Argentina Fil: Altamirano, Lorena. Sociedad Neurológica Argentina; Argentina Fil: Amores, Mirtha Graciela. Sociedad Neurológica Argentina; Argentina Fil: Bertotti, Alicia Cristina. Sociedad Neurológica Argentina; Argentina Fil: Cueto, Alicia. Sociedad Neurológica Argentina; Argentina Fil: Díaz Livadiotis, Guillermo. Sociedad Neurológica Argentina; Argentina Fil: Di Egidio, Mariana. Sociedad Neurológica Argentina; Argentina Fil: Doumic, Javier. Sociedad Neurológica Argentina; Argentina Fil: Dubrovsky, Alberto. Sociedad Neurológica Argentina; Argentina Fil: Fulgenzi, Ernesto. Sociedad Neurológica Argentina; Argentina Fil: Lautre, Andrea. Sociedad Neurológica Argentina; Argentina Fil: Losavio, Adriana Silvia. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Houssay. Instituto de Investigaciones Médicas. Universidad de Buenos Aires. Facultad de Medicina. Instituto de Investigaciones Médicas; Argentina Fil: Marchesone, Cintia. Sociedad Neurológica Argentina; Argentina Fil: Martinez Alvarez, Mariana. Sociedad Neurológica Argentina; Argentina Fil: Mazia, Claudio Gabriel. Sociedad Neurológica Argentina; Argentina Fil: Melano, Raúl. Sociedad Neurológica Argentina; Argentina Fil: Orellano, Anabel. Sociedad Neurológica Argentina; Argentina Fil: Pagano, Miguel Angel. Sociedad Neurológica Argentina; Argentina Fil: Pardal, Ana Maria. Sociedad Neurológica Argentina; Argentina Fil: Pirra, Laura. Sociedad Neurológica Argentina; Argentina Fil: Politei, Juan. Sociedad Neurológica Argentina; Argentina Fil: Reisin, Ricardo. Sociedad Neurológica Argentina; Argentina Fil: Rey, Roberto. Sociedad Neurológica Argentina; Argentina Fil: Rodriguez, Gabriel. Sociedad Neurológica Argentina; Argentina Fil: Rugiero, Marcelo. Sociedad Neurológica Argentina; Argentina Fil: Yorio, Alberto. Sociedad Neurológica Argentina; Argentina

Published
2006

33. TERAPEUTICAS INTERVENCIONISTAS PARA EL ACCIDENTE CEREBROVASCULAR ISQUEMICO.

Author

Rabadan, Alejandra T., Sposato, Luciano, and Mazia, Claudio

Abstract

Copyright of Medicina (Buenos Aires) is the property of Medicina (Buenos Aires) and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)

Published
2010

34. Adverse Outcome of Early Recurrent Ischemic Stroke Secondary to Atrial Fibrillation after Repeated Systemic Thrombolysis

Author

A. Sposato, Luciano, Salutto, Valeria, E. Beratti, Diego, Monti, Paula, M. Riccio, Patricia, and Mazia, Claudio

Abstract

Background. Recurrent ischemic stroke is associated with adverse neurological outcome in patients with atrial fibrillation. There is very scarce information regarding the neurological outcome of atrial fibrillation patients undergoing repeated systemic thrombolysis after early recurrent ischemic stroke. Clinical Case and Discussion. We describe a case of a 76-year-old woman with known paroxysmal atrial fibrillation who was admitted because of an acute right middle cerebral artery ischemic stroke and who underwent repeated systemic thrombolysis within 110 hours. The patient underwent systemic thrombolysis after the first ischemic stroke with almost complete neurological recovery. On the fourth day after treatment, an acute left middle cerebral artery ischemic stroke was diagnosed and she was treated with full-dose intravenous recombinant tissue plasminogen activator. A hemorrhagic transformation of the left middle cerebral artery infarction was noted on follow-up cranial computed tomographic scans. The patient did not recover from the second cerebrovascular event and died 25 days after admission. Conclusion. To the best of our knowledge, this is the second case reporting the adverse neurological outcome of a patient with diagnosis of atrial fibrillation undergoing repeated systemic thrombolysis after early recurrent ischemic stroke. Our report represents a contribution to the scarce available evidence suggesting that repeated systemic thrombolysis for recurrent ischemic stroke should be avoided.

Published
2013
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36. [Argentine consensus on late-onset Pompe's disease].

Author

Dubrovsky A, Fulgenzi E, De Vito EL, Barroso F, Berardo A, Bettini M, Binaghi D, Calabrese E, Carlés D, Chaves M, Chloca F, Conti E, Corderi J, Di Gennaro F, Ferradás N, Jáuregui A, Lubieniecki F, Mazia C, Medina M, Pirra L, Politei J, Reisin R, Rosa AL, Rugiero M, Salutto V, Schenone A, Sussini M, and Taratuto AL

Subjects
Age of Onset, Argentina, Glycogen Storage Disease Type II complications, Humans, Glycogen Storage Disease Type II diagnosis, Glycogen Storage Disease Type II therapy
Abstract

Pompe's disease (PD) is an infrequent metabolic autosomic recessive disorder produced by the lack or deficiency of the acid alpha-glucosidase lysosomal enzyme in tissues of involved individuals. Delayed-onset PD is considered whenever symptoms onset start after one year of age. We present an update of the recommendations for the management of delayed-onset PD, taking as reference the guidelines from the Argentine Consensus for diagnosis, treatment and follow-up of PD published in 2013. The present consensus gathered several experts in PD in the areas of internal medicine, laboratory diagnosis, neuropathology, pulmonology, nutrition, neurology, metabolic and neuromuscular disorders as well as rehabilitation to perform an update of the literature of delayed-onset PD, with special attention on relevant information published within the last 4 years. The entire working group approved the final version of the consensus. Each participant provided a declaration of conflict of interest. As a result, it is an update of the previous Argentine PD Consensus with focus on the delayed-onset presentation of the disease. Being such infrequent disorder, available data were rather limited and thus, the recommendations represent expert opinions.

Published
2018

37. [Update on interventional treatment of acute ischemic stroke].

Author

Rabadán AT, Sposato L, and Mazia C

Subjects
Angioplasty, Decompressive Craniectomy methods, Endarterectomy, Carotid, Humans, Thrombolytic Therapy methods, Tomography, X-Ray Computed, Stroke therapy
Abstract

New options have been developed for the prevention and treatment of acute ischemic stroke in the last 20 years, such as carotid endarterectomy and intravenous thrombolysis with tissue plasminogen activator. Scientific evidence has supported their use in developed countries, while there is an evident delay in their use among emerging countries. Other promising modalities require the conclusion of ongoing randomized, controlled trials. Malignant middle or carotid cerebral artery infarction accounts for 10 to 15% ischemic strokes and constitutes a devastating event associated with high morbidity and mortality. Decompressive craniectomy seems to be an effective and safe approach for rapidly lowering intracranial pressure. Although randomized trials are lacking, there is enough evidence to support this surgical procedure in appropriately selected patients.

Published
2010

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