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1. Efficacy and safety of maintenance intravenous immunoglobulin in generalized myasthenia gravis patients with acetylcholine receptor antibodies: A multicenter, double-blind, placebo-controlled trial.

Author

Bril, Vera, Berkowicz, Tomasz, Szczudlik, Andrzej, Nicolle, Michael, Bednarik, Josef, Hon, Petr, Vaitkus, Antanas, Vu, Tuan, Rozsa, Csilla, Magnus, Tim, Panczel, Gyula, Toomsoo, Toomas, Pasnoor, Mamatha, Mozaffar, Tahseen, Freimer, Miriam, Reuner, Ulrike, Vécsei, László, Souayah, Nizar, Levine, Todd, Pascuzzi, Robert, Dalakas, Marinos, Rivner, Michael, Griffin, Rhonda, Coll, Montse, and Mondou, Elsa

Subjects
autoimmune disease, intravenous immunoglobulin, myasthenia gravis, neuromuscular disease, Humans, Myasthenia Gravis, Immunoglobulins, Intravenous, Male, Female, Double-Blind Method, Middle Aged, Adult, Receptors, Cholinergic, Aged, Treatment Outcome, Autoantibodies, Immunologic Factors, Activities of Daily Living, Prospective Studies
Abstract

INTRODUCTION/AIMS: Prospective, randomized, controlled trials of intravenous immunoglobulin (IVIG) maintenance therapy in myasthenia gravis (MG) are lacking. In this trial, we evaluated the safety and efficacy of caprylate/chromatography-purified IVIG; (IGIV-C) in patients with generalized MG undergoing standard care. METHODS: Sixty-two patients enrolled in this phase 2, multicenter, international, randomized trial (1:1 IGIV-C [2 g/kg loading dose; 1 g/kg every 3 weeks through week 21] or placebo). Efficacy was assessed by changes in Quantitative MG (QMG) score at week 24 versus baseline (primary endpoint) and percentage of patients with clinical improvement in QMG, MG Composite (MGC), and MG-Activities of Daily Living (MG-ADL) scores (secondary endpoints). Safety assessments reported all adverse events (AEs). RESULTS: The change in QMG at 24 weeks was -5.1 for IGIV-C and -3.1 for placebo (p = .187). Seventy percent of patients in the IGIV-C group had improvement in MG-ADL (≥2-point decrease) versus 40.6% in the placebo group (p = .025). Patients showing clinical improvement in QMG and MGC (≥3-point decrease) were 70.0% for IGIV-C versus 59.4% for placebo (p = .442) and 60.0% for IGIV-C versus 53.1% for placebo (p = .610). IGIV-C was well tolerated; serious AEs were similar between arms. Three of four MG exacerbations requiring hospitalizations occurred in the IGIV-C arm with one death. DISCUSSION: Several efficacy parameters showed numerical results greater than those seen in the placebo group. This was a small study and may have been underpowered to see significant differences. Additional studies may be warranted to fully determine the efficacy of IVIG maintenance therapy in MG.

Published
2025

3. Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial

Author

Grinzinger, Susanne, Wanschitz, Julia, Seifert-Held, Thomas, Claeys, Kristl G., Baets, Jonathan, Remiche, Gauthier, Bissay, Véronique, Dubuisson, Nicolas, Delstanche, Stephanie, Tarnev, Ivaylo, Genov, Krasimir, Tsvetanov, Plamen, Milanov, Ivan, Zhao, Chongbo, Bu, Bitao, Yu, Xuefan, Li, Wei, Jiang, Haishan, Da, Yuwei, Lu, Zuneng, Liang, Hui, Guo, Fuqiang, Li, Zunbo, Zou, Zhangyu, Hong, Daojun, Yang, Huan, Guo, Junhong, Shi, Jianquan, Tu, Jianglong, He, Dian, Wang, Yiqi, Ding, Jing, Zhang, Yali, Zhao, Yuanqi, Xu, Renxi, Yue, Yunhua, Guo, Aihong, Wang, Yuzhong, Talab, Radomir, Harbo, Thomas, Sindrup, Soeren, De Seze, Jérôme, Sacconi, Sabrina, Péréon, Yann, Echaniz-Laguna, Andoni, Magy, Laurent, Nicolas, Guillaume, Taithe, Frédéric, Cassereau, Julien, Debs, Rabab, Shakarishvili, Roman, Tsiskaridze, Alexander, Mania, Maka, Janelidze, Marina, Janelidze, Tamar, Schroeter, Michael, Skripuletz, Thomas, Lee, De-Hyung, Klehmet, Juliane, Hotter, Benjamin, Hoffmann, Olaf, Baum, Petra, Zschuentzsch, Jana, Pitarokoili, Kalliopi, Stettner, Mark, Bereczki, Dániel, Pánczél, Gyula, Abraham, Alon, Dori, Amir, Lampl, Yair, Manganelli, Fiore, Morino, Stefania, Padovani, Alessandro, Siciliano, Gabriele, Schenone, Angelo, Magri, Francesca, Mazzeo, Anna, Giannini, Fabio, Sorbi, Sandro, Chiò, Adriano, Kuwahara, Motoi, Okuno, Tatsusada, Okamoto, Tomoko, Kokubun, Norito, Nishiyama, Kazutoshi, Kaida, Kenichi, Bokuda, Kota, Katsuno, Masahisa, Yabe, Ichiro, Saji, Etsuji, Yokota, Takanori, Hatanaka, Yuki, Nakahara, Jin, Sugimoto, Takamichi, Tanaka, Fumiaki, Tomita, Satoshi, Yamano, Yoshihisa, Hayashi, Tomohiro, Yamazaki, Hiroki, Tokashiki, Takashi, Horiuchi, Kazuhiro, Karelis, Guntis, Eftimov, Filip, Banach, Marta, Chyrchel-Paszkiewicz, Urszula, Kochanowicz, Jan, Selmaj, Krzysztof, Zielinski, Tomasz, Banaszkiewicz, Krzysztof, Mitrea, Dan, Scutaru-Kadar, Ana-Maria, Axelerad, Any, Stuchevskaya, Fatima, Boyko, Alexey, Khabirov, Farit, Trushnikova, Tatiana, Goncharova, Zoya, Suponeva, Natalia, Dorogov, Nikolay, Yakupov, Eduard, Raicevic, Ranko, Miletic Drakulic, Svetlana, Cabrera Serrano, Macarena, Muñoz Blanco, Jose Luis, Guerrero Sola, Antonio, Aguera Morales, Eduardo, Diaz Marin, Carmen, Juntas Morales, Raúl, Yeh, Jiann-Horng, Sung, Jia-Ying, Huang, Han-Wei, Tsai, Nai-Wen, Guo, Yuh-Cherng, Chao, Chi-Chao, Ro, Long-Sun, Sengun, Ihsan, Terzi, Murat, Alpaydin Baslo, Sezin, Koç, Filiz, Necdet Karli, Hamdi, Shulga, Olga, Smolko, Dmytro, Doroshenko, Oleksandr, Tomakh, Nataliya, Kyrychenko, Alla, Seliuk, Olga, Kalbus, Oleksandr, Skrypchenko, Iryna, Novakovska, Olha, Carod-Artal, Francisco Javier, Rinaldi, Simon, Brennan, Kathryn, Ellis, Simon, Carr, Aisling, Matthews, Emma, Traub, Rebecca, Mozaffar, Tahseen, Elliott, Matthew, Bhavaraju-Sanka, Ratna, Nance, Christopher, Levine, Todd, Lisak, Robert, Pasnoor, Mamatha, Pulley, Michael, Roy, Bhaskar, Govindarajan, Raghav, Sahagian, Gregory, Khella, Sami, Jacob, Daniel, Kushlaf, Hani, Sivakumar, Kumaraswamy, Melamed, Isaac, Sharma, Khema, Quick, Adam, Ubogu, Eroboghene, Lacomis, David, Isa, Arnaldo, Brannagan, Thomas, Chen, Shan, Katz, Jonathan, Feinberg, Marc, Pavlakis, Pantelis, Lange, Dale, Gudesblatt, Mark, Tandan, Rup, Gable, Karissa, Rivner, Michael, Barnes, Benjamin, Luke, Donna, Mahuwala, Zabeen, Macwan, Samir, Kwon, Patrick, Scott, James, Altamimi, Sadiq, Sabharwal, Priyanka, Allen, Jeffrey A, Lin, Jie, Basta, Ivana, Dysgaard, Tina, Eggers, Christian, Guptill, Jeffrey T, Gwathmey, Kelly G, Hewamadduma, Channa, Hofman, Erik, Hussain, Yessar M, Kuwabara, Satoshi, Le Masson, Gwendal, Leypoldt, Frank, Chang, Ting, Lipowska, Marta, Lowe, Murray, Lauria, Giuseppe, Querol, Luis, Simu, Mihaela-Adriana, Suresh, Niraja, Tse, Anissa, Ulrichts, Peter, Van Hoorick, Benjamin, Yamasaki, Ryo, Lewis, Richard A, and van Doorn, Pieter A

Published
2024
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5. Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial

Author

Granit, Volkan, Steele, Julie, Levy, Wendy, Paredes, Maria Elena, Hernandez, Jessica, Bilsker, Martin, Szacka, Katarzyna, Ronert, Adam, Jablońska, Dorota, Łuczak, Alina Zuzanna, Chaverri, Delia, Janse van Mantgem, Mark R, Bunte, Tommy M, Broere, Bianca, de Fockert, Arianne, Sanchez-Tejerina, Daniel, Landabaso, Carmen, Calvo, Andrea, Moglia, Cristina, Manera, Umberto, Canosa, Antonio, Vasta, Rosario, Salamone, Paolina, Fuda, Giuseppe, DeMarco, Giovanni, Casale, Federico, ME Forsberg, Karin, Winroth, Ivar, Almgren Stenberg, Erica, Holmgren, Monica, Amador, Maria del Mar, Lenglet, Timothee, Querin, Giorgia, Coudoin, Sylvie, Pavlakis, Pantelis, Holzberg, Shara, Sideri, Riccardo, Marinou, Kalliopi, Czarnecki, Maciej, Ługiewicz, Renata, Biel-Czarnecka, Marta, Boczkowska, Marcelina, Schotte, Caroline, Vynckier, Jan, Van Daele, Sien, Claeys, Thomas, Delmotte, Koen, Swinnen, Bart, Serrien, Anouk, D'Hondt, Ann, Lamaire, Nikita, Debien, Elisa, Jones, Sarah, Vachon, Chris, Grogan, James, Solorzano, Guillermo, Crowell, Allison, Rakocevic, Goran, Wagoner, Mary, Alma, Osmanovic, Flavia, Wiehler, Sonja, Körner, Olivia, Schreiber-Katz, Camilla, Wohnrade, Anastasia, Sarikidi, Carola, Kassebaum, Chantal, Fischer, Adamo, Ashley, Turcotte, Nicole, Duncan, Jessie, Turner, Ivone, Elman, Lauren, Massie, Rami, Berube, Maxime, Saunders, Natalie, Salmon, Kristiana, Foucher, Juliette, Agessandro, Abrahao, Shirley, Pham, Jahan, Mookshah, Phung, Liane, Statland, Jeffrey, Jawdat, Omar, Dimachkie, Mazen, Pasnoor, Mamatha, Farmakidis, Constantine, Heim, Andrew, Lillig, Katie, Lackey, Alyssa, Weber, Markus, Kurz, Martina, Levine, Todd, Benatar, Michael, Hansen, Thomas, Rom, Dror, Geist, Marie A, Blaettler, Thomas, Camu, William, Kuzma-Kozakiewicz, Magdalena, van den Berg, Leonard H, Morales, Raul Juntas, Chio, Adriano, Andersen, Peter M, Pradat, Pierre-Francois, Lange, Dale, Van Damme, Philip, Mora, Gabriele, Grudniak, Mariusz, Elliott, Matthew, Petri, Susanne, Olney, Nicholas, Ladha, Shafeeq, Goyal, Namita A, Meyer, Thomas, Hanna, Michael G, Quinn, Colin, Genge, Angela, Zinman, Lorne, Jabari, Duaa, Shoesmith, Christen, Ludolph, Albert C, Neuwirth, Christoph, Nations, Sharon, Shefner, Jeremy M, Turner, Martin R, Wuu, Joanne, Bennett, Richard, Dang, Hoang, and Sundgreen, Claus

Published
2024
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7. Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial

Author

Dimachkie, Mazen, Statland, Jeffrey, Pasnoor, Mamatha, Jawdat, Omar, Heim, Andrew, Ciersdorff, Ali, Sasidharan, Sandhya, Currence, Melissa, Levine, Todd, Otutoa, Rebecca, Cooper, Angelina, Mozaffar, Tahseen, Habib, Ali, Cauchi, Jonathan, Ung, Shannon, Mathew, Veena, Hernandez, Isela, Gibson, Summer, Bromberg, Mark, Mahoney, Kyle, Neate, Crystal, Janecki, Teresa, Papadakis, Mike, Freimer, Miriam, Kaschalk, MacKenzie, Heintzman, Sarah, Wicklund, Matthew, Baines, Brenna, Vareldzis, Alexa, Hyslop, Emily, Blume, Brianna, Ciafaloni, Emma, Luebbe, Elizabeth, Eichinger, Katy, Martens, William, Gregory, Stephanie, Janciuras, Joanne, Amato, Anthony, Doughty, Christopher, Roe, Kristen, Flynn, Patricia, Russo, Emily, Lloyd, Thomas, Albayda, Jemima, Tiniakou, Eleni, Thomas, Simone, Jones, Sarah, Solorzano, Guillermo, Elliott, Matthew, Burns, Ted, Crowell, Allison, Eggleston, Deborah, Wagoner, Mary, Shaibani, Aziz, Oates, Chantae, Machado, Pedro, Hanna, Michael, Greensmith, Linda, Ahmed, Mhoriam, Vivekanandam, Vinojini, Appleby, Matthew, Ransley, George, Eshun, Edwin Eshun, Skorupinska, Iwona, Germain, Louise, Laxa, Ana Marie, Pontes, Joana Roca, Bellin, Anna, Anifowoshe, Dolapo, Machado, Pedro M, McDermott, Michael P, Blaettler, Thomas, Sundgreen, Claus, Amato, Anthony A, Gibson, Summer B, Jones, Sarah M, Levine, Todd D, Lloyd, Thomas E, Shaibani, Aziz I, Rosholm, Anders, Carstensen, Tim Dehli, Bonefeld, Karen, Jørgensen, Anders Nørkær, Phonekeo, Karina, Heim, Andrew J, Herbelin, Laura, Barohn, Richard J, Hanna, Michael G, and Dimachkie, Mazen M

Published
2023
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8. Epidemiological evidence for a hereditary contribution to myasthenia gravis: a retrospective cohort study of patients from North America

Author

Green, Joshua D, Barohn, Richard J, Bartoccion, Emanuela, Benatar, Michael, Blackmore, Derrick, Chaudhry, Vinay, Chopra, Manisha, Corse, Andrea, Dimachkie, Mazen M, Evoli, Amelia, Florence, Julaine, Freimer, Miriam, Howard, James F, Jiwa, Theresa, Kaminski, Henry J, Kissel, John T, Koopman, Wilma J, Lipscomb, Bernadette, Maestri, Michelanglo, Marino, Mariapaola, Massey, Janice M, McVey, April, Mezei, Michelle M, Muppidi, Srikanth, Nicolle, Michael W, Oger, Joel, Pascuzzi, Robert M, Pasnoor, Mamatha, Pestronk, Alan, Provenzano, Carlo, Ricciardi, Roberta, Richman, David P, Rowin, Julie, Sanders, Donald B, Siddiqi, Zaeem, Soloway, Aimee, Wolfe, Gil I, Wulf, Charlie, Drachman, Daniel B, and Traynor, Bryan J

Subjects
Genetics, Neurodegenerative, Clinical Research, Myasthenia Gravis, Autoimmune Disease, Rare Diseases, Neurosciences, 2.1 Biological and endogenous factors, Aetiology, Autoantibodies, Humans, North America, Receptors, Cholinergic, Retrospective Studies, epidemiology, neuromuscular disease, genetics, neurology, neurogenetics, Clinical Sciences, Public Health and Health Services, Other Medical and Health Sciences
Abstract

ObjectivesTo approximate the rate of familial myasthenia gravis and the coexistence of other autoimmune disorders in the patients and their families.DesignRetrospective cohort study.SettingClinics across North America.ParticipantsThe study included 1032 patients diagnosed with acetylcholine receptor antibody (AChR)-positive myasthenia gravis.MethodsPhenotype information of 1032 patients diagnosed with AChR-positive myasthenia gravis was obtained from clinics at 14 centres across North America between January 2010 and January 2011. A critical review of the epidemiological literature on the familial rate of myasthenia gravis was also performed.ResultsAmong 1032 patients, 58 (5.6%) reported a family history of myasthenia gravis. A history of autoimmune diseases was present in 26.6% of patients and in 28.4% of their family members.DiscussionThe familial rate of myasthenia gravis was higher than would be expected for a sporadic disease. Furthermore, a high proportion of patients had a personal or family history of autoimmune disease. Taken together, these findings suggest a genetic contribution to the pathogenesis of myasthenia gravis.

Published
2020

9. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study

Author

Sembinelli, Dylan, Teitelbaum, Jeanne, Nicolle, Michael, Bernard, Emilien, Svahn, Juliette, Spinazzi, Marco, Stojkovic, Tanya, Demeret, Sophie, Weiss, Nicolas, Le Guennec, Loïc, Messai, Sihame, Tranchant, Christine, Nadaj-Pakleza, Aleksandra, Chanson, Jean-Baptiste, Suliman, Muhtadi, Zaidi, Leila, Tard, Celine, Lecointe, Peggy, Zschüntzsch, Jana, Schmidt, Jens, Glaubitz, Stefanie, Zeng, Rachel, Scholl, Matthias, Kowarik, Markus, Ziemann, Ulf, Krumbholz, Markus, Martin, Pascal, Ruschil, Christoph, Dünschede, Jutta, Kemmner, Roswitha, Rumpel, Natalie, Berger, Benjamin, Totzeck, Andreas, Hagenacker, Tim, Stolte, Benjamin, Iorio, Raffaele, Evoli, Amelia, Falso, Silvia, Antozzi, Carlo, Frangiamore, Rita, Vanoli, Fiammetta, Rinaldi, Elena, Deguchi, Kazushi, Minami, Naoya, Nagane, Yuriko, Suzuki, Yasushi, Ishida, Sayaka, Suzuki, Shigeaki, Nakahara, Jin, Nagaoka, Astushi, Yoshimura, Shunsuke, Konno, Shingo, Tsuya, Youko, Uzawa, Akiyuki, Kubota, Tomoya, Takahashi, Masanori, Okuno, Tatsusada, Murai, Hiroyuki, Gilhus, Nils Erik, Boldingh, Marion, Rønning, Tone Hakvåg, Chyrchel-Paszkiewicz, Urszula, Kumor, Klaudiusz, Zielinski, Tomasz, Banaszkiewicz, Krzysztof, Błaż, Michał, Kłósek, Agata, Świderek-Matysiak, Mariola, Szczudlik, Andrzej, Paśko, Aneta, Szczechowski, Lech, Banach, Marta, Ilkowski, Jan, Kapetanovic Garcia, Solange, Ortiz Bagan, Patricia, Belén Cánovas Segura, Ana, Turon Sans, Joana, Vidal Fernandez, Nuria, Cortes Vicente, Elena, Rodrigo Armenteros, Patricia, Ashraghi, Mohammad, Cavey, Ana, Haslam, Liam, Emery, Anna, Liow, Kore, Yegiaian, Sharon, Barboi, Alexandru, Vazquez, Rosa Maria, Lennon, Joshua, Pascuzzi, Robert M, Bodkin, Cynthia, Guingrich, Sandra, Comer, Adam, Bromberg, Mark, Janecki, Teresa, Saba, Sami, Tellez, Marco, Elsheikh, Bakri, Freimer, Miriam, Heintzman, Sarah, Govindarajan, Raghav, Guptill, Jeffrey, Massey, Janice M, Juel, Vern, Gonzalez, Natalia, Habib, Ali A, Mozaffar, Tahseen, Korb, Manisha, Goyal, Namita, Machemehl, Hannah, Manousakis, Georgios, Allen, Jeffrey, Harper, Emily, Farmakidis, Constantine, Saavedra, Lilli, Dimachkie, Mazen, Pasnoor, Mamatha, Akhter, Salma, Beydoun, Said, McIlduff, Courtney, Nye, Joan, Roy, Bhaskar, Munro Sheldon, Bailey, Nowak, Richard, Barnes, Benjamin, Rivner, Michael, Suresh, Niraja, Shaw, Jessica, Harvey, Brittany, Lam, Lucy, Thomas, Nikki, Chopra, Manisha, Traub, Rebecca E, Jones, Sarah, Wagoner, Mary, Smajic, Sejla, Aly, Radwa, Katz, Jonathan, Chen, Henry, Miller, Robert G, Jenkins, Liberty, Khan, Shaida, Khatri, Bhupendra, Sershon, Lisa, Pavlakis, Pantelis, Holzberg, Shara, Li, Yuebing, Caristo, Irys B, Marquardt, Robert, Hastings, Debbie, Rube, Jacob, Lisak, Robert P, Choudhury, Aparna, Ruzhansky, Katherine, Sachdev, Amit, Shin, Susan, Bratton, Joan, Fetter, Mary, McKinnon, Naya, McKinnon, Jonathan, Sissons-Ross, Laura, Sahu, Amos, Distad, B Jane, Howard, James F, Jr, Bresch, Saskia, Genge, Angela, Hewamadduma, Channa, Hinton, John, Hussain, Yessar, Juntas-Morales, Raul, Kaminski, Henry J, Maniaol, Angelina, Mantegazza, Renato, Masuda, Masayuki, Sivakumar, Kumaraswamy, Śmiłowski, Marek, Utsugisawa, Kimiaki, Vu, Tuan, Weiss, Michael D, Zajda, Małgorzata, Boroojerdi, Babak, Brock, Melissa, de la Borderie, Guillemette, Duda, Petra W, Lowcock, Romana, Vanderkelen, Mark, and Leite, M Isabel

Published
2023
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10. Zilucoplan in immune-mediated necrotising myopathy: a phase 2, randomised, double-blind, placebo-controlled, multicentre trial

Author

Amato, Anthony A., Benveniste, Olivier, Biliciler, Suur, Chinoy, Hector, Dimachkie, Mazen M., Edmundson, Christyn, Freimer, Miriam, Geraci, Anthony, Hussain, Yessar, Machado, Pedro, Mammen, Andrew L., Mozaffar, Tahseen, Soltanzadeh, Payam, Suresh, Niraja, van der Kooi, Anneke, Allenbach, Yves, Appleby, Matthew, Barohn, Richard J, Champtiaux, Nicolas, Doughty, Christopher, Farias, Jerrica, Farmakidis, Constantine, Habib, Ali A., Karam, Chafic, Lilleker, James, Lorusso, Samantha, Pasnoor, Mamatha, Pinal-Fernandez, Iago, Querin, Giorgia, Raaphorst, Joost, Ransley, George, Saba, Sami, Sheikh, Kazim, Snedden, Andrew, Statland, Jeffrey, Vu, Tuan, Mammen, Andrew L, Amato, Anthony A, Dimachkie, Mazen M, Lilleker, James B, Boroojerdi, Babak, Vanderkelen, Mark, Delicha, Eumorphia Maria, Koendgen, Harold, Farzaneh-Far, Ramin, Duda, Petra W, and Sayegh, Camil

Published
2023
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11. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

Author

Behin, Anthony, Boentert, Matthias, Carvalho, Gerson, Chahin, Nizar, Charrow, Joel, Deegan, Patrick, Durmus Tekce, Hacer, Duval, Fanny, Genge, Angela, Gutmann, Ludwig, Henderson, Robert D, Hennermann, Julia B, Hiwot, Tarekegn, Hughes, Derralynn, Karaa, Amel, Karam, Chafic, Kautzky-Willer, Alexandra, Komaki, Hirofumi, Laforet, Pascal, Longo, Nicola, Malinova, Vera, Maré, Ricardo, Maxit, Clarisa, Mengel, Eugen, Moggio, Maurizio Gualtiero, Molnár, Mária Judit, Mongini, Tiziana Enrica, Nadaj-Pakleza, Aleksandra, Nascimento Osorio, Andres, Noury, Jean-Baptiste, Oliveira, Acary Souza Bulle, Parman, Yesim, Pena, Loren, Remiche, Gauthier, Sciacco, Monica, Shieh, Perry B, Smith, Cheryl, Stulnig, Thomas, Taithe, Frederic, Tard, Céline, Tarnopolsky, Mark, Vorgerd, Matthias, Whitley, Chester, Young, Peter, Alonso-Pérez, Jorge, Altemus, Patricia, Aubé-Nathier, Anne-Catherine, Avelar, Jennifer B, Bailey, Carrie, Bekircan-Kurt, Can Ebru, Billy, Jenny, Boschi, Silvia, Brown, Kathryn E, Carrera Garcia, Laura, Chase, Lauren, Cirne, Hamilton, Danjoux, Loïc, Davion, Jean-Baptiste, DeArmey, Stephanie, Fedotova, Ekaterina, Gandolfo, Eve, Grosz, Zoltan, Guellec, Dewi, Guettsches, Anne-Katrin, Guglieri, Michela, Hatcher, Erin, Helms, Sina, Hufgard-Leitner, Miriam, Klyushnikov, Sergey A., Langton, Jacqui, Linková, Lenka, Mavroudakis, Nicolas, Mazurová, Stella, Mori, Madoka, Müller-Miny, Louisa, Musumeci, Olimpia, Nance, Christopher S, Natera-de Benito, Daniel, Neel, Robert, Niizawa, Gabriela A, Noll, Lauren, Ortega, Erik, Pasnoor, Mamatha, Pautot, Vivien, Potulska-Chromik, Anna, Pugliese, Alessia, Questienne, Claire, Ramos Lopes, Margarida, Reyes-Leiva, David, Riedl, Michaela, Rugiero, Marcelo Francisco, Salort-Campana, Emmanuelle, Sgobbi Souza, Paulo Victor, Sole, Guilhem, Solera, Luca, Souto Lopes, Suzara, Specht, Sabine, Statland, Jeffrey, Swenson, Andrea, Tan, Chong Yew, Tizon, Sónia, van der Beek, N A M E, van Kooten, Harmke A., Wencel, Marie, Wenninger, Stephan, Zagnoli, Fabien, Diaz-Manera, Jordi, Kishnani, Priya S, Kushlaf, Hani, Ladha, Shafeeq, Mozaffar, Tahseen, Straub, Volker, Toscano, Antonio, van der Ploeg, Ans T, Berger, Kenneth I, Clemens, Paula R, Chien, Yin-Hsiu, Day, John W, Illarioshkin, Sergey, Roberts, Mark, Attarian, Shahram, Borges, Joao Lindolfo, Bouhour, Francoise, Choi, Young Chul, Erdem-Ozdamar, Sevim, Goker-Alpan, Ozlem, Kostera-Pruszczyk, Anna, Haack, Kristina An, Hug, Christopher, Huynh-Ba, Olivier, Johnson, Judith, Thibault, Nathan, Zhou, Tianyue, Dimachkie, Mazen M, and Schoser, Benedikt

Published
2021
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13. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial

Author

De Bleecker, Jan L., De Koning, Kathy, De Mey, Katrien, De Pue, Annelien, Mercelis, Rudolf, Wyckmans, Maren, Vinck, Caroline, Wagemaekers, Linda, Baets, Jonathan, Ng, Eduardo, Shabanpour, Jafar, Daniyal, Lubna, Mannan, Shabber, Katzberg, Hans D., Genge, Angela, Siddiqi, Zaeem, Junkerová, Jana, Horakova, Jana, Reguliova, Katerina, Tyblova, Michaela, Jurajdova, Ivana, Novakova, Iveta, Jakubikova, Michala, Pitha, Jiri, Vohanka, Stanislav, Havelkova, Katerina, Horak, Tomas, Bednarik, Josef, Horakova, Mageda, Meisel, Andreas, Remstedt, Dike, Heibutzki, Claudia, Kohler, Siegfried, Gerischer, Lea, Hoffman, Sarah, Stascheit, Frauke, Vissing, John, Zafirakos, Lizzie, Khatri, Kuldeep Kumar, Autzen, Anne, Godtfeldt Stemmerik, Mads Peter, Andersen, Henning, Attarian, Shahram, Salort-Campana, Emmanuelle, Delmont, Emilien, Grapperon, Aude-Marie, Kouton, Ludivine, Tsiskaridze, Alexander, Rózsa, Csilla, Jakab, Gedeonne Margo, Toth, Szilvia, Szabo, Gyorgyi, Bors, David, Szabo, Eniko, Campanella, Angela, Vanoli, Fiammetta, Frangiamore, Rita, Antozzi, Carlo, Bonanno, Silvia, Maggi, Lorenzo, Giossi, Riccardo, Saccà, Francesco, Marsili, Angela, Pane, Chiara, Puorro, Giorgia, Reia, Antonio, Antonini, Giovanni, Alfieri, Girolamo, Morino, Stefania, Garibaldi, Matteo, Fionda, Laura, Leonardi, Luca, Konno, Shingo, Uzawa, Akiyuki, Sakuma, Kaoru, Watanabe, Chiho, Ozawa, Yukiko, Yasuda, Manato, Onishi, Yosuke, Samukawa, Makoto, Tsuda, Tomoko, Suzuki, Yasushi, Ishida, Sayaka, Watanabe, Genya, Takahashi, Masanori, Nakamura, Hiroko, Sugano, Erina, Kubota, Tomoya, Imai, Tomihiro, Suzuki., Mari, Mori, Ayako, Yamamoto, Daisuke, Ikeda, Kazuna, Hisahara, Shin, Masuda, Masayuki, Takaki, Miki, Minemoto, Kanako, Ido, Nobuhiro, Naito, Makiko, Okubo, Yoshihiko, Sugimoto, Takamichi, Takematsu, Yuka, Kamei, Ayumi, Shimizu, Mihiro, Naito, Hiroyuki, Nomura, Eiichi, Van Heur, Marjolein, Peters, Anne-Marie, Tannemaat, Martijn, Ruiter, Annabel, Keene, Kevin, Halas, Marek, Szczudlik, Andrzej, Pinkosz, Marta, Frasinska, Monika, Zwolinska, Grazyna, Kostera-Pruszczyk, Anna, Golenia, Aleksandra, Szczudlik, Piotr, Szczechowski, Lech, Pasko, Aneta, Poverennova, Irina, Urtaeva, Lubov, Kuznetsova, Nadezhda, Romanova, Tatiana, Nadezhda, Malkova, Lapochka, Elena, Korobko, Denis, Vergunova, Ilona, Melnikova, Anna, Bulatova, Ekaterina, Antipenko, Elena, Basta, Ivana, Bozovic, Ivo, Lavrnic, Dragana, Stojanovic, Vidosava Rakocevic, Beydoun, Said, Akhter, Salma, Malekniazi, Ali, Darki, Leila, Pimentel, Norianne, Cannon, Victoria, Chopra, Manisha, Traub, Rebecca, Mozaffar, Tahseen, Hernandez, Isela, Turner, Ivonne, Habib, Ali, Goyal, Namita, Kak, Manisha, Velasquez, Erik, Lam, Lucy, Suresh, Niraja, Farias, Jerrica, Jones, Sarah, Wagoner, Mary, Eggleston, Debbie, Bertorini, Tulio, Benzel, Cindy, Henegar, Robert, Pillai, Rekha, Bharavaju-Sanka, Ratna, Paiz, Carolyn, Jackson, Carlayne, Ruzhansky, Katherine, Dimitrova, Diana, Visser, Amy, Chahin, Nizar, Levine, Todd, Lisak, Robert, Jia, Kelly, Mada, Flicia, Bernitsas, Evanthia, Pasnoor, Mamatha, Roath, Katherine, Colgan, Samantha, Currence, Melissa, Heim, Andrew, Barohn, Richard, Dimachkie, Mazen, Statland, Jeffrey, Jawdat, Omar, Jabari, Duaa, Farmakidis, Constantine, Gilchrist, James, Li, Yuebing, Caristo, Irys, Hastings, Debbie, Morren, John Anthony, Weiss, Michael, Muppidi, Srikanth, Nguyen, Tia, Welsh, Lesly, So, Yuen, Goyal, Neelam, Pulley, Michael, Bailey, Cathy, Quraishi, Zubair, Berger, Alan, Sahagian, Gregory, Camberos, Yasmin, Frishberg, Benjamin, Howard, James F, Jr, Bril, Vera, Vu, Tuan, Karam, Chafic, Peric, Stojan, Margania, Temur, Murai, Hiroyuki, Bilinska, Malgorzata, Shakarishvili, Roman, Smilowski, Marek, Guglietta, Antonio, Ulrichts, Peter, Vangeneugden, Tony, Utsugisawa, Kimiaki, Verschuuren, Jan, and Mantegazza, Renato

Published
2021
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14. Inclusion Body Myositis Treatment with Celution Processed Adipose Derived Regenerative Cells (P8-11.005)

Author

Heim, Andrew, primary, Soder, Rupal, additional, Bhavsar, Dhaval, additional, Russo, Ali, additional, Agbas, Abdulbaki, additional, Kosa, Edina, additional, Pasnoor, Mamatha, additional, Jawdat, Omar, additional, Varon, Matthew, additional, Farmakidis, Constantine, additional, Chandrashekhar, Swathy, additional, and Dimachkie, Mazen, additional

Published
2024
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17. A randomized controlled trial of methotrexate for patients with generalized myasthenia gravis.

Author

Pasnoor, Mamatha, He, Jianghua, Herbelin, Laura, Burns, Ted M, Nations, Sharon, Bril, Vera, Wang, Annabel K, Elsheikh, Bakri H, Kissel, John T, Saperstein, David, Shaibani, J Aziz, Jackson, Carlayne, Swenson, Andrea, Howard, James F, Goyal, Namita, David, William, Wicklund, Matthew, Pulley, Michael, Becker, Mara, Mozaffar, Tahseen, Benatar, Michael, Pazcuzzi, Robert, Simpson, Ericka, Rosenfeld, Jeffrey, Dimachkie, Mazen M, Statland, Jeffrey M, Barohn, Richard J, and Methotrexate in MG Investigators of the Muscle Study Group

Subjects
Methotrexate in MG Investigators of the Muscle Study Group, Humans, Myasthenia Gravis, Methotrexate, Prednisone, Receptors, Cholinergic, Immunosuppressive Agents, Autoantibodies, Treatment Outcome, Severity of Illness Index, Area Under Curve, Double-Blind Method, Adult, Aged, Aged, 80 and over, Middle Aged, Canada, United States, Female, Male, Clinical Trials and Supportive Activities, Rare Diseases, Autoimmune Disease, Neurosciences, Clinical Research, Orphan Drug, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, Clinical Sciences, Cognitive Sciences, Neurology & Neurosurgery
Abstract

ObjectiveTo determine the steroid-sparing effect of methotrexate (MTX) in patients with symptomatic generalized myasthenia gravis (MG).MethodsWe performed a 12-month multicenter, randomized, double-blind, placebo-controlled trial of MTX 20 mg orally every week vs placebo in 50 acetylcholine receptor antibody-positive patients with MG between April 2009 and August 2014. The primary outcome measure was the prednisone area under the dose-time curve (AUDTC) from months 4 to 12. Secondary outcome measures included 12-month changes of the Quantitative Myasthenia Gravis Score, the Myasthenia Gravis Composite Score, Manual Muscle Testing, the Myasthenia Gravis Quality of Life, and the Myasthenia Gravis Activities of Daily Living.ResultsFifty-eight patients were screened and 50 enrolled. MTX did not reduce the month 4-12 prednisone AUDTC when compared to placebo (difference MTX - placebo: -488.0 mg, 95% confidence interval -2,443.4 to 1,467.3, p = 0.26); however, the average daily prednisone dose decreased in both groups. MTX did not improve secondary measures of MG compared to placebo over 12 months. Eight participants withdrew during the course of the study (1 MTX, 7 placebo). There were no serious MTX-related adverse events. The most common adverse event was nonspecific pain (19%).ConclusionsWe found no steroid-sparing benefit of MTX in MG over 12 months of treatment, despite being well-tolerated. This study demonstrates the challenges of conducting clinical trials in MG, including difficulties with recruitment, participants improving on prednisone alone, and the need for a better understanding of outcome measure variability for future clinical trials.Classification of evidenceThis study provides Class I evidence that for patients with generalized MG MTX does not significantly reduce the prednisone AUDTC over 12 months of therapy.

Published
2016

18. A Genome-Wide Association Study of Myasthenia Gravis

Author

Renton, Alan E, Pliner, Hannah A, Provenzano, Carlo, Evoli, Amelia, Ricciardi, Roberta, Nalls, Michael A, Marangi, Giuseppe, Abramzon, Yevgeniya, Arepalli, Sampath, Chong, Sean, Hernandez, Dena G, Johnson, Janel O, Bartoccioni, Emanuela, Scuderi, Flavia, Maestri, Michelangelo, Gibbs, J Raphael, Errichiello, Edoardo, Chiò, Adriano, Restagno, Gabriella, Sabatelli, Mario, Macek, Mark, Scholz, Sonja W, Corse, Andrea, Chaudhry, Vinay, Benatar, Michael, Barohn, Richard J, McVey, April, Pasnoor, Mamatha, Dimachkie, Mazen M, Rowin, Julie, Kissel, John, Freimer, Miriam, Kaminski, Henry J, Sanders, Donald B, Lipscomb, Bernadette, Massey, Janice M, Chopra, Manisha, Howard, James F, Koopman, Wilma J, Nicolle, Michael W, Pascuzzi, Robert M, Pestronk, Alan, Wulf, Charlie, Florence, Julaine, Blackmore, Derrick, Soloway, Aimee, Siddiqi, Zaeem, Muppidi, Srikanth, Wolfe, Gil, Richman, David, Mezei, Michelle M, Jiwa, Theresa, Oger, Joel, Drachman, Daniel B, and Traynor, Bryan J

Subjects
Rare Diseases, Genetics, Neurosciences, Autoimmune Disease, Myasthenia Gravis, Clinical Research, Human Genome, 2.1 Biological and endogenous factors, Aetiology, Adult, Age of Onset, CTLA-4 Antigen, Case-Control Studies, Female, Gene Frequency, Genetic Predisposition to Disease, Genome-Wide Association Study, Genotype, HLA-DQ alpha-Chains, Humans, Male, Middle Aged, Polymorphism, Single Nucleotide, United States, Clinical Sciences, Cognitive Sciences, Neurology & Neurosurgery
Abstract

ImportanceMyasthenia gravis is a chronic, autoimmune, neuromuscular disease characterized by fluctuating weakness of voluntary muscle groups. Although genetic factors are known to play a role in this neuroimmunological condition, the genetic etiology underlying myasthenia gravis is not well understood.ObjectiveTo identify genetic variants that alter susceptibility to myasthenia gravis, we performed a genome-wide association study.Design, setting, and participantsDNA was obtained from 1032 white individuals from North America diagnosed as having acetylcholine receptor antibody-positive myasthenia gravis and 1998 race/ethnicity-matched control individuals from January 2010 to January 2011. These samples were genotyped on Illumina OmniExpress single-nucleotide polymorphism arrays. An independent cohort of 423 Italian cases and 467 Italian control individuals were used for replication.Main outcomes and measuresWe calculated P values for association between 8,114,394 genotyped and imputed variants across the genome and risk for developing myasthenia gravis using logistic regression modeling. A threshold P value of 5.0×10(-8) was set for genome-wide significance after Bonferroni correction for multiple testing.ResultsIn the overall case-control cohort, we identified association signals at CTLA4 (rs231770; P=3.98×10(-8); odds ratio, 1.37; 95% CI, 1.25-1.49), HLA-DQA1 (rs9271871; P=1.08×10(-8); odds ratio, 2.31; 95% CI, 2.02-2.60), and TNFRSF11A (rs4263037; P=1.60×10(-9); odds ratio, 1.41; 95% CI, 1.29-1.53). These findings replicated for CTLA4 and HLA-DQA1 in an independent cohort of Italian cases and control individuals. Further analysis revealed distinct, but overlapping, disease-associated loci for early- and late-onset forms of myasthenia gravis. In the late-onset cases, we identified 2 association peaks: one was located in TNFRSF11A (rs4263037; P=1.32×10(-12); odds ratio, 1.56; 95% CI, 1.44-1.68) and the other was detected in the major histocompatibility complex on chromosome 6p21 (HLA-DQA1; rs9271871; P=7.02×10(-18); odds ratio, 4.27; 95% CI, 3.92-4.62). Association within the major histocompatibility complex region was also observed in early-onset cases (HLA-DQA1; rs601006; P=2.52×10(-11); odds ratio, 4.0; 95% CI, 3.57-4.43), although the set of single-nucleotide polymorphisms was different from that implicated among late-onset cases.Conclusions and relevanceOur genetic data provide insights into aberrant cellular mechanisms responsible for this prototypical autoimmune disorder. They also suggest that clinical trials of immunomodulatory drugs related to CTLA4 and that are already Food and Drug Administration approved as therapies for other autoimmune diseases could be considered for patients with refractory disease.

Published
2015

19. 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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21. 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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22. Leg amyotrophic diplegia: prevalence and pattern of weakness at US neuromuscular centers.

Author

Dimachkie, Mazen, Muzyka, Iryna, Katz, Jonathan, Jackson, Carlayne, Wang, Yunxia, McVey, April, Dick, Arthur, Pasnoor, Mamatha, Mozaffar, M, Xiao-Song, Z, Kissel, John, Ensrud, E, Rosenfeld, Jeffrey, and Barohn, Richard

Subjects
Academic Medical Centers, Age of Onset, Disease Progression, Female, Humans, Leg, Male, Middle Aged, Muscular Atrophy, Spinal, Prevalence, Retrospective Studies, United States
Abstract

OBJECTIVE: To identify the frequency of leg amyotrophic diplegia (LAD) at a US academic center, describe the pattern of weakness, and provide comparative data from 8 additional major US academic institutions. BACKGROUND: LAD is a leg onset variant of progressive muscular atrophy (PMA). LAD weakness is confined to the legs for at least 2 years, and there are no upper motor neuron signs. DESIGN/METHODS: We present a retrospective chart review of 24 patients with the LAD presentation from the University of Kansas Medical Center ( n = 8 cases) and from 8 US academic institutions (n = 16 cases). RESULTS: Of the 318 subjects identified in the University of Kansas Medical Center Neuromuscular Research Database, 82% (260 subjects) had amyotrophic lateral sclerosis (ALS), 1.9% (6) had familial ALS, 6.6% (21) had primary lateral sclerosis, and 9.2% (29) had lower motor neuron (LMN) disease. Of these 29 cases, 16 had PMA, 5 had brachial amyotrophic diplegia, while 8 had LAD. The mean LAD age of onset was 58 years with a male/female ratio of 3/1. Onset was asymmetric in 7/8. We identified a pelviperoneal pattern of weakness (sparing of knee extension and/or ankle plantar flexion) in 4 cases and distal predominant weakness in 3 cases. All patients had electrodiagnostic findings consistent with motor neuron disease confined to the lower extremities. We present LAD disease duration and survival data from 8 major academic neuromuscular centers. At last follow-up, weakness progressed to involve the arms in 6/24 LAD cases and of these 6 cases, 2 patients died from progression to typical ALS. From onset of symptoms, mean survival in LAD is 87 months, with 92% of cases being alive. CONCLUSIONS/RELEVANCE: The natural history of LAD differs from typical forms of ALS and PMA. LAD is a slowly progressive disorder that accounts for a fourth of LMN disease cases. An asymmetric pelviperoneal pattern of weakness should heighten the suspicion for LAD.

Published
2013

23. Leg Amyotrophic Diplegia

Author

Dimachkie, Mazen M, Muzyka, Iryna M, Katz, Jonathan S, Jackson, Carlayne, Wang, Yunxia, McVey, April L, Dick, Arthur, Pasnoor, Mamatha, Mozaffar, M Tahseen, Xiao-Song, Z, Kissel, John T, Ensrud, E, Rosenfeld, Jeffrey, and Barohn, Richard J

Subjects
Biomedical and Clinical Sciences, Neurosciences, Clinical Sciences, Neurodegenerative, ALS, Rare Diseases, Brain Disorders, Clinical Research, Aetiology, 2.1 Biological and endogenous factors, Neurological, Academic Medical Centers, Age of Onset, Disease Progression, Female, Humans, Leg, Male, Middle Aged, Muscular Atrophy, Spinal, Prevalence, Retrospective Studies, United States, Neurology & Neurosurgery, Clinical sciences
Abstract

ObjectiveTo identify the frequency of leg amyotrophic diplegia (LAD) at a US academic center, describe the pattern of weakness, and provide comparative data from 8 additional major US academic institutions.BackgroundLAD is a leg onset variant of progressive muscular atrophy (PMA). LAD weakness is confined to the legs for at least 2 years, and there are no upper motor neuron signs.Design/methodsWe present a retrospective chart review of 24 patients with the LAD presentation from the University of Kansas Medical Center ( n = 8 cases) and from 8 US academic institutions (n = 16 cases).ResultsOf the 318 subjects identified in the University of Kansas Medical Center Neuromuscular Research Database, 82% (260 subjects) had amyotrophic lateral sclerosis (ALS), 1.9% (6) had familial ALS, 6.6% (21) had primary lateral sclerosis, and 9.2% (29) had lower motor neuron (LMN) disease. Of these 29 cases, 16 had PMA, 5 had brachial amyotrophic diplegia, while 8 had LAD. The mean LAD age of onset was 58 years with a male/female ratio of 3/1. Onset was asymmetric in 7/8. We identified a pelviperoneal pattern of weakness (sparing of knee extension and/or ankle plantar flexion) in 4 cases and distal predominant weakness in 3 cases. All patients had electrodiagnostic findings consistent with motor neuron disease confined to the lower extremities. We present LAD disease duration and survival data from 8 major academic neuromuscular centers. At last follow-up, weakness progressed to involve the arms in 6/24 LAD cases and of these 6 cases, 2 patients died from progression to typical ALS. From onset of symptoms, mean survival in LAD is 87 months, with 92% of cases being alive.Conclusions/relevanceThe natural history of LAD differs from typical forms of ALS and PMA. LAD is a slowly progressive disorder that accounts for a fourth of LMN disease cases. An asymmetric pelviperoneal pattern of weakness should heighten the suspicion for LAD.

Published
2013

25. Long-term safety, tolerability, and efficacy of efgartigimod in patients with generalized myasthenia gravis: Concluding analyses from the ADAPT+ study

Author

Bril, Vera, primary, Pasnoor, Mamatha, additional, Karam, Chafic, additional, Peric, Stojan, additional, De Bleecker, Jan L., additional, Murai, Hiroyuki, additional, Meisel, Andreas, additional, Beydoun, Said, additional, Vu, Tuan, additional, Ulrichts, Peter, additional, Van Hoorick, Benjamin, additional, T'Joen, Caroline, additional, Kaprielian, Roger, additional, Utsugisawa, Kimiaki, additional, Verschuuren, Jan, additional, Mantegazza, Renato, additional, and Howard, James F., additional

Published
2023
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26. Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial

Author

Machado, Pedro M, primary, McDermott, Michael P, additional, Blaettler, Thomas, additional, Sundgreen, Claus, additional, Amato, Anthony A, additional, Ciafaloni, Emma, additional, Freimer, Miriam, additional, Gibson, Summer B, additional, Jones, Sarah M, additional, Levine, Todd D, additional, Lloyd, Thomas E, additional, Mozaffar, Tahseen, additional, Shaibani, Aziz I, additional, Wicklund, Matthew, additional, Rosholm, Anders, additional, Carstensen, Tim Dehli, additional, Bonefeld, Karen, additional, Jørgensen, Anders Nørkær, additional, Phonekeo, Karina, additional, Heim, Andrew J, additional, Herbelin, Laura, additional, Barohn, Richard J, additional, Hanna, Michael G, additional, Dimachkie, Mazen M, additional, Dimachkie, Mazen, additional, Statland, Jeffrey, additional, Pasnoor, Mamatha, additional, Jawdat, Omar, additional, Heim, Andrew, additional, Ciersdorff, Ali, additional, Sasidharan, Sandhya, additional, Currence, Melissa, additional, Levine, Todd, additional, Otutoa, Rebecca, additional, Cooper, Angelina, additional, Habib, Ali, additional, Cauchi, Jonathan, additional, Ung, Shannon, additional, Mathew, Veena, additional, Hernandez, Isela, additional, Gibson, Summer, additional, Bromberg, Mark, additional, Mahoney, Kyle, additional, Neate, Crystal, additional, Janecki, Teresa, additional, Papadakis, Mike, additional, Kaschalk, MacKenzie, additional, Heintzman, Sarah, additional, Baines, Brenna, additional, Vareldzis, Alexa, additional, Hyslop, Emily, additional, Blume, Brianna, additional, Luebbe, Elizabeth, additional, Eichinger, Katy, additional, Martens, William, additional, Gregory, Stephanie, additional, Janciuras, Joanne, additional, Amato, Anthony, additional, Doughty, Christopher, additional, Roe, Kristen, additional, Flynn, Patricia, additional, Russo, Emily, additional, Lloyd, Thomas, additional, Albayda, Jemima, additional, Tiniakou, Eleni, additional, Thomas, Simone, additional, Jones, Sarah, additional, Solorzano, Guillermo, additional, Elliott, Matthew, additional, Burns, Ted, additional, Crowell, Allison, additional, Eggleston, Deborah, additional, Wagoner, Mary, additional, Shaibani, Aziz, additional, Oates, Chantae, additional, Machado, Pedro, additional, Hanna, Michael, additional, Greensmith, Linda, additional, Ahmed, Mhoriam, additional, Vivekanandam, Vinojini, additional, Appleby, Matthew, additional, Ransley, George, additional, Eshun, Edwin Eshun, additional, Skorupinska, Iwona, additional, Germain, Louise, additional, Laxa, Ana Marie, additional, Pontes, Joana Roca, additional, Bellin, Anna, additional, and Anifowoshe, Dolapo, additional

Published
2023
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27. Database Evaluation for Muscle and Nerve Diseases - DEMAND: An academic neuromuscular coding system

Author

Pasnoor, Mamatha, primary, Khan, Saud, additional, Jackson, Carlayne E., additional, Kissel, John, additional, Wolfe, Gil I., additional, Nations, Sharon P., additional, Trivedi, Jaya R., additional, Singer, Mike A., additional, Elliott, Jeffrey L., additional, Vernino, Steven, additional, Mummaneni, Reddiah Babu, additional, Herbelin, Laura, additional, Saperstein, David S., additional, Gronseth, Gary, additional, McVey, April L., additional, Dimachkie, Mazen M., additional, and Barohn, Richard J., additional

Published
2023
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30. Long-term safety, tolerability, and efficacy of efgartigimod (ADAPT+): interim results from a phase 3 open-label extension study in participants with generalized myasthenia gravis.

Author

Howard Jr., James F., Bril, Vera, Tuan Vu, Karam, Chafic, Peric, Stojan, De Bleecker, Jan L., Hiroyuki Murai, Meisel, Andreas, Beydoun, Said R., Pasnoor, Mamatha, Guglietta, Antonio, Van Hoorick, Benjamin, Steeland, Sophie, T'joen, Caroline, Kimiaki Utsugisawa, Verschuuren, Jan, and Mantegazza, Renato

Subjects
MYASTHENIA gravis, INTRAVENOUS therapy, MUSCLE weakness, FC receptors, IMMUNOGLOBULIN G
Abstract

Objective: ADAPT+ assessed the long-term safety, tolerability, and efficacy of efgartigimod in adult participants with generalized myasthenia gravis (gMG). Methods: ADAPT+ was an open-label, single-arm, multicenter, up to 3-year extension of the pivotal phase 3 ADAPT study. Efgartigimod was administered in treatment cycles of 4 intravenous infusions (one 10 mg/kg infusion per week). Initiation of subsequent treatment cycles was individualized based on clinical evaluation. Safety endpoints included incidence and severity of adverse events. Efficacy endpoints assessed disease severity using Myasthenia Gravis-Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores. Results: As of January 2022, 151 participants had rolled over to ADAPT+ and 145 had received ≥1 dose of efgartigimod, of whom, 111 (76.6%) were AChR-Ab+ and 34 (23.4%) were AChR-Ab-. Mean study duration (treatment plus follow-up) was 548 days, and participants received up to 17 treatment cycles, corresponding to 217.6 participant-years of exposure. In the overall population, 123 (84.8%) participants reported ≥1 treatment-emergent adverse event; most frequent were headache (36 [24.8%]), COVID-19 (22 [15.2%]), and nasopharyngitis (20 [13.8%]). Clinically meaningful improvement (CMI) in mean MG-ADL and QMG scores was seen as early as 1 week following the first infusion across multiple cycles in AChR-Ab+ and AChR-Ab-participants. Maximal MG-ADL and QMG improvements aligned with onset and magnitude of total IgG and AChR-Ab reductions. For AChR-Ab+ participants at any time point in each of the first 10 treatment cycles, more than 90% had a maximum reduction of ≥2 points (CMI) in MG-ADL total score; across the 7 cycles in which QMG was measured, 69.4% to 91.3% of participants demonstrated a maximum reduction of ≥3 points (CMI) in QMG total score. Many participants demonstrated improvements well beyond CMI thresholds. In AChR-Ab+ participants with ≥1 year of combined follow-up between ADAPT and ADAPT+, mean number of annualized cycles was 4.7 per year (median [range] 5.0 [0.5-7.6]). Conclusion: Results of ADAPT+ corroborate the substantial clinical improvements seen with efgartigimod in ADAPT and support its long-term safety, tolerability, and efficacy, as well as an individualized dosing regimen for treatment of gMG. [ABSTRACT FROM AUTHOR]

Published
2024
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31. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study

Author

Howard, James F, primary, Bresch, Saskia, additional, Genge, Angela, additional, Hewamadduma, Channa, additional, Hinton, John, additional, Hussain, Yessar, additional, Juntas-Morales, Raul, additional, Kaminski, Henry J, additional, Maniaol, Angelina, additional, Mantegazza, Renato, additional, Masuda, Masayuki, additional, Sivakumar, Kumaraswamy, additional, Śmiłowski, Marek, additional, Utsugisawa, Kimiaki, additional, Vu, Tuan, additional, Weiss, Michael D, additional, Zajda, Małgorzata, additional, Boroojerdi, Babak, additional, Brock, Melissa, additional, de la Borderie, Guillemette, additional, Duda, Petra W, additional, Lowcock, Romana, additional, Vanderkelen, Mark, additional, Leite, M Isabel, additional, Sembinelli, Dylan, additional, Teitelbaum, Jeanne, additional, Nicolle, Michael, additional, Bernard, Emilien, additional, Svahn, Juliette, additional, Spinazzi, Marco, additional, Stojkovic, Tanya, additional, Demeret, Sophie, additional, Weiss, Nicolas, additional, Le Guennec, Loïc, additional, Messai, Sihame, additional, Tranchant, Christine, additional, Nadaj-Pakleza, Aleksandra, additional, Chanson, Jean-Baptiste, additional, Suliman, Muhtadi, additional, Zaidi, Leila, additional, Tard, Celine, additional, Lecointe, Peggy, additional, Zschüntzsch, Jana, additional, Schmidt, Jens, additional, Glaubitz, Stefanie, additional, Zeng, Rachel, additional, Scholl, Matthias, additional, Kowarik, Markus, additional, Ziemann, Ulf, additional, Krumbholz, Markus, additional, Martin, Pascal, additional, Ruschil, Christoph, additional, Dünschede, Jutta, additional, Kemmner, Roswitha, additional, Rumpel, Natalie, additional, Berger, Benjamin, additional, Totzeck, Andreas, additional, Hagenacker, Tim, additional, Stolte, Benjamin, additional, Iorio, Raffaele, additional, Evoli, Amelia, additional, Falso, Silvia, additional, Antozzi, Carlo, additional, Frangiamore, Rita, additional, Vanoli, Fiammetta, additional, Rinaldi, Elena, additional, Deguchi, Kazushi, additional, Minami, Naoya, additional, Nagane, Yuriko, additional, Suzuki, Yasushi, additional, Ishida, Sayaka, additional, Suzuki, Shigeaki, additional, Nakahara, Jin, additional, Nagaoka, Astushi, additional, Yoshimura, Shunsuke, additional, Konno, Shingo, additional, Tsuya, Youko, additional, Uzawa, Akiyuki, additional, Kubota, Tomoya, additional, Takahashi, Masanori, additional, Okuno, Tatsusada, additional, Murai, Hiroyuki, additional, Gilhus, Nils Erik, additional, Boldingh, Marion, additional, Rønning, Tone Hakvåg, additional, Chyrchel-Paszkiewicz, Urszula, additional, Kumor, Klaudiusz, additional, Zielinski, Tomasz, additional, Banaszkiewicz, Krzysztof, additional, Błaż, Michał, additional, Kłósek, Agata, additional, Świderek-Matysiak, Mariola, additional, Szczudlik, Andrzej, additional, Paśko, Aneta, additional, Szczechowski, Lech, additional, Banach, Marta, additional, Ilkowski, Jan, additional, Kapetanovic Garcia, Solange, additional, Ortiz Bagan, Patricia, additional, Belén Cánovas Segura, Ana, additional, Turon Sans, Joana, additional, Vidal Fernandez, Nuria, additional, Cortes Vicente, Elena, additional, Rodrigo Armenteros, Patricia, additional, Ashraghi, Mohammad, additional, Cavey, Ana, additional, Haslam, Liam, additional, Emery, Anna, additional, Liow, Kore, additional, Yegiaian, Sharon, additional, Barboi, Alexandru, additional, Vazquez, Rosa Maria, additional, Lennon, Joshua, additional, Pascuzzi, Robert M, additional, Bodkin, Cynthia, additional, Guingrich, Sandra, additional, Comer, Adam, additional, Bromberg, Mark, additional, Janecki, Teresa, additional, Saba, Sami, additional, Tellez, Marco, additional, Elsheikh, Bakri, additional, Freimer, Miriam, additional, Heintzman, Sarah, additional, Govindarajan, Raghav, additional, Guptill, Jeffrey, additional, Massey, Janice M, additional, Juel, Vern, additional, Gonzalez, Natalia, additional, Habib, Ali A, additional, Mozaffar, Tahseen, additional, Korb, Manisha, additional, Goyal, Namita, additional, Machemehl, Hannah, additional, Manousakis, Georgios, additional, Allen, Jeffrey, additional, Harper, Emily, additional, Farmakidis, Constantine, additional, Saavedra, Lilli, additional, Dimachkie, Mazen, additional, Pasnoor, Mamatha, additional, Akhter, Salma, additional, Beydoun, Said, additional, McIlduff, Courtney, additional, Nye, Joan, additional, Roy, Bhaskar, additional, Munro Sheldon, Bailey, additional, Nowak, Richard, additional, Barnes, Benjamin, additional, Rivner, Michael, additional, Suresh, Niraja, additional, Shaw, Jessica, additional, Harvey, Brittany, additional, Lam, Lucy, additional, Thomas, Nikki, additional, Chopra, Manisha, additional, Traub, Rebecca E, additional, Jones, Sarah, additional, Wagoner, Mary, additional, Smajic, Sejla, additional, Aly, Radwa, additional, Katz, Jonathan, additional, Chen, Henry, additional, Miller, Robert G, additional, Jenkins, Liberty, additional, Khan, Shaida, additional, Khatri, Bhupendra, additional, Sershon, Lisa, additional, Pavlakis, Pantelis, additional, Holzberg, Shara, additional, Li, Yuebing, additional, Caristo, Irys B, additional, Marquardt, Robert, additional, Hastings, Debbie, additional, Rube, Jacob, additional, Lisak, Robert P, additional, Choudhury, Aparna, additional, Ruzhansky, Katherine, additional, Sachdev, Amit, additional, Shin, Susan, additional, Bratton, Joan, additional, Fetter, Mary, additional, McKinnon, Naya, additional, McKinnon, Jonathan, additional, Sissons-Ross, Laura, additional, Sahu, Amos, additional, and Distad, B Jane, additional

Published
2023
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33. Mills’ Disease Variant (P3-8.012)

Author

Moussallem, Rasha, primary, Jawdat, Omar, additional, Jabari, Duaa, additional, Pasnoor, Mamatha, additional, Dimachkie, Mazen, additional, and Statland, Jeffery, additional

Published
2023
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35. Phase 2 trial in acetylcholine receptor antibody‐positive myasthenia gravis of transition from intravenous to subcutaneous immunoglobulin: The MGSCIg study

Author

Pasnoor, Mamatha, primary, Bril, Vera, additional, Levine, Todd, additional, Trivedi, Jaya, additional, Silvestri, Nicholas J., additional, Phadnis, Milind, additional, Katzberg, Hans D., additional, Saperstein, David S., additional, Wolfe, Gil I., additional, Herbelin, Laura, additional, Higgs, Kiley, additional, Heim, Andrew J., additional, Statland, Jeffrey M., additional, Barohn, Richard J., additional, and Dimachkie, Mazen M., additional

Published
2023
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36. Zilucoplan in immune-mediated necrotising myopathy: a phase 2, randomised, double-blind, placebo-controlled, multicentre trial

Author

Mammen, Andrew L, primary, Amato, Anthony A, additional, Dimachkie, Mazen M, additional, Chinoy, Hector, additional, Hussain, Yessar, additional, Lilleker, James B, additional, Pinal-Fernandez, Iago, additional, Allenbach, Yves, additional, Boroojerdi, Babak, additional, Vanderkelen, Mark, additional, Delicha, Eumorphia Maria, additional, Koendgen, Harold, additional, Farzaneh-Far, Ramin, additional, Duda, Petra W, additional, Sayegh, Camil, additional, Benveniste, Olivier, additional, Amato, Anthony A., additional, Biliciler, Suur, additional, Dimachkie, Mazen M., additional, Edmundson, Christyn, additional, Freimer, Miriam, additional, Geraci, Anthony, additional, Machado, Pedro, additional, Mammen, Andrew L., additional, Mozaffar, Tahseen, additional, Soltanzadeh, Payam, additional, Suresh, Niraja, additional, van der Kooi, Anneke, additional, Appleby, Matthew, additional, Barohn, Richard J, additional, Champtiaux, Nicolas, additional, Doughty, Christopher, additional, Farias, Jerrica, additional, Farmakidis, Constantine, additional, Habib, Ali A., additional, Karam, Chafic, additional, Lilleker, James, additional, Lorusso, Samantha, additional, Pasnoor, Mamatha, additional, Querin, Giorgia, additional, Raaphorst, Joost, additional, Ransley, George, additional, Saba, Sami, additional, Sheikh, Kazim, additional, Snedden, Andrew, additional, Statland, Jeffrey, additional, and Vu, Tuan, additional

Published
2023
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41. Long-term Safety and Efficacy of Efgartigimod in Patients With Generalized Myasthenia Gravis: Interim Results of the ADAPT+ Study

Author

Howard, James, primary, Bril, Vera, additional, Vu, Tuan, additional, Karam, Chafic, additional, Peric, Stojan, additional, De Bleecker, Jan, additional, Murai, Hiroyuki, additional, Meisel, Andreas, additional, Beydoun, Said, additional, Pasnoor, Mamatha, additional, Guglietta, Antonio, additional, Ulrichts, Peter, additional, T'joen, Caroline, additional, Brauer, Edward, additional, Utsugisawa, Kimiaki, additional, Verschuuren, Jan, additional, and Mantegazza, Renato, additional

Published
2022
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44. Rituximab as treatment for anti-MuSK myasthenia gravis: Multicenter blinded prospective review

Author

Hehir, Michael K., Hobson-Webb, Lisa D., Benatar, Michael, Barnett, Carolina, Silvestri, Nicholas J., Howard, James F., Jr, Howard, Diantha, Visser, Amy, Crum, Brian A., Nowak, Richard, Beekman, Rachel, Kumar, Aditya, Ruzhansky, Katherine, Chen, I-Hweii Amy, Pulley, Michael T., LaBoy, Shannon M., Fellman, Melissa A., Greene, Shane M., Pasnoor, Mamatha, and Burns, Ted M.

Published
2017
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46. Hyaluronidase‐facilitated subcutaneous immunoglobulin 10% as maintenance therapy for chronic inflammatory demyelinating polyradiculoneuropathy: The ADVANCE‐CIDP 1 randomized controlled trial.

Author

Bril, Vera, Hadden, Robert D. M., Brannagan, Thomas H., Bar, Michal, Chroni, Elisabeth, Rejdak, Konrad, Rivero, Alberto, Andersen, Henning, Latov, Norman, Levine, Todd, Pasnoor, Mamatha, Sacconi, Sabrina, Souayah, Nizar, Anderson‐Smits, Colin, Duff, Kim, Greco, Erin, Hasan, Shabbir, Li, Zhaoyang, Yel, Leman, and Ay, Hakan

Subjects
THERAPEUTIC use of immunoglobulins, DRUG efficacy, IMMUNOGLOBULINS, INTRAVENOUS therapy, CONFIDENCE intervals, TREATMENT duration, HEALTH outcome assessment, HEALTH surveys, RANDOMIZED controlled trials, PLACEBOS, TREATMENT effectiveness, GUILLAIN-Barre syndrome, GLYCOSIDASES, BLIND experiment, QUESTIONNAIRES, DESCRIPTIVE statistics, RESEARCH funding, SUBCUTANEOUS infusions, STATISTICAL sampling, PATIENT safety, EVALUATION
Abstract

Background and Aims: ADVANCE‐CIDP 1 evaluated facilitated subcutaneous immunoglobulin (fSCIG; human immunoglobulin G 10% with recombinant human hyaluronidase) efficacy and safety in preventing chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) relapse. Methods: ADVANCE‐CIDP 1 was a phase 3, double‐blind, placebo‐controlled trial conducted at 54 sites in 21 countries. Eligible adults had definite or probable CIDP and adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scores of 0–7 (inclusive), and received stable intravenous immunoglobulin (IVIG) for ≥12 weeks before screening. After stopping IVIG, patients were randomized 1:1 to fSCIG 10% or placebo for 6 months or until relapse/discontinuation. fSCIG 10% was administered at the same dose (or matching placebo volume) and interval as pre‐randomization IVIG. The primary outcome was patient proportion experiencing CIDP relapse (≥1‐point increase in adjusted INCAT score from pre‐subcutaneous treatment baseline) in the modified intention‐to‐treat population. Secondary outcomes included time to relapse and safety endpoints. Results: Overall, 132 patients (mean age 54.4 years, 56.1% male) received fSCIG 10% (n = 62) or placebo (n = 70). CIDP relapse was reduced with fSCIG 10% versus placebo (n = 6 [9.7%; 95% confidence interval 4.5%, 19.6%] vs n = 22 [31.4%; 21.8%, 43.0%], respectively; absolute difference: −21.8% [−34.5%, −7.9%], p =.0045). Relapse probability was higher with placebo versus fSCIG 10% over time (p =.002). Adverse events (AEs) were more frequent with fSCIG 10% (79.0% of patients) than placebo (57.1%), but severe (1.6% vs 8.6%) and serious AEs (3.2% vs 7.1%) were less common. Interpretation: fSCIG 10% more effectively prevented CIDP relapse than placebo, supporting its potential use as maintenance CIDP treatment. [ABSTRACT FROM AUTHOR]

Published
2023
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48. Activity for Diabetic Polyneuropathy (ADAPT): study design and protocol for a 2-site randomized controlled trial

Author

Kluding, Patricia M., Singleton, J. Robinson, Pasnoor, Mamatha, Dimachkie, Mazen M., Barohn, Richard J., Smith, A. Cordon, and Marcus, Robin L.

Subjects
Care and treatment, Development and progression, Health aspects, Lifestyles -- Health aspects, Quality of life -- Health aspects, Diabetic neuropathies -- Development and progression -- Care and treatment
Abstract

Approximately 30 million Americans have diabetes, half of whom will develop diabetic peripheral neuropathy (DPN). (1) Diabetic peripheral neuropathy causes reduced mobility and quality of life due to pain, sensory [...], Background. Half of all patients with diabetes develop diabetic peripheral neuropathy (DPN), a complication leading to reduced mobility and quality of life. Although there are no proven pharmacologic approaches to reduce DPN risk or slow its progression, evidence suggests that physical activity may improve symptoms and enhance peripheral nerve regeneration. Objective. The aim of the study will be to determine the impact of an intense lifestyle intervention on neuropathy progression and quality of life in individuals with DPN. Design. The study will be a randomized controlled trial. Setting. The study will be conducted at 2 academic medical centers. Participants. The participants will be 140 individuals with type 2 diabetes and mild to moderate DPN. Intervention. The intervention group will receive 18 months of supervised exercise training, actigraphy-based counseling to reduce sedentary behavior, and individualized dietary counseling. Control group participants will receive diet and activity counseling at baseline and at 9 months. Measurements. The primary outcomes are neuropathy progression as measured by intraepidermal nerve fiber density in a distal thigh skin biopsy and the Norfolk Quality of Life-Diabetic Neuropathy score. Secondary outcomes include pain, gait, balance, and mobility measures. Limitations. Due to the combined intervention approach, this protocol will not be able to determine which intervention components influence outcomes. There also may be difficulty with participant attrition during the 18-month study intervention. Conclusions. The Activity for Diabetic Polyneuropathy (ADAPT) protocol resulted from a collaboration between physical therapists and neurologist researchers that includes as primary outcomes both a quality-of-life measure (NQOL-DN) and a physiologic biomarker (IENFD). It has the potential to demonstrate that an intensive lifestyle intervention may be a sustainable, clinically effective approach for people with DPN that improves patient outcomes and can have an immediate impact on patient care and future clinical trials.

Published
2017
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