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1,927 results on '"Sokal, Etienne"'

1. Type of calcineurin inhibitor and long-term outcomes following liver transplantation in patients with primary biliary cholangitis – an ELTR study

Author

Mirza, Darius, Heneghan, Michael, Lodge, Peter, Oniscu, Gabriel, Thorburn, Douglas, Allison, Michael, Pratschke, Johann, Manas, Derek, Bennet, William, Line, Pal-Dag, Hot, Emir, Zieniewicz, Krzysztof, Ericzon, Bo Goran, Fronek, Jiri, Klempnauer, Jurgen L., Rasmussen, Allan, Romagnoli, Renato, Nemec, Petr, Nordin, Arno, Paul, Andreas, De Simone, Paolo, Porte, R.J., Berlakovich, Gabriela, Cherqui, Daniel, Pirenne, Jacques, Sokal, Etienne, Rossi, Giorgio, Candinas, Daniel, Bachellier, Philippe, Rummo, Oleg, Boudjema, Karim, Mrzljak, Anna, Soubrane, Olivier, Metselaar, Herold, Schneeberger, Stefan, Navarro, Francis, Berney, Thierry, Duvoux, Christophe, Colledan, Michele, De Carlis, Luciano, Boillot, Olivier, Hardwigsen, Jean, Pruvot, Francois Rene, Suc, Bertrand, Vivarelli, Marco, Clavien, Pierre Alain, Lang, Hauke, Kosieradzki, Maciej, Berrevoet, Frederik, Heyd, Bruno, Cescon, Matteo, Chiche, Laurence, Kochs, Eberhard, Baccarani, Umberto, Detry, Olivier, Bartels, Michael, Rossi, Massimo, Scatton, Olivier, Papanikolaou, Vasileios, Alwayn, Ian, Schemmer, Peter, Senninger, N., Ducerf, Christian, Di Benedetto, Fabrizio, Tisone, Giuseppe, Nadalin, Silvio, Mathe, Zoltan, Ribnikar, Marija, Settmacher, Utz, Becker, Thomas, Silva, Nuno, Daniel, Jorge, Popescu, Irinel, Lucidi, Valerio, Bechstein, Wolf O., Decaens, Thomas, Gugenheim, Jean, Gruttadauria, Salvatore, Zamboni, Frausto, Zeytunlu, Murat, Kalff, Jorg C., Vali, Toomas, Tokat, Yaman, Klar, Ernst, Janek, Julius, Kilic, Murat, Katzarov, Krum, Fisher, Lutz, Buc, Emmanuel, Castagneto, Marco, Unek, Tarkan, Spassov, Lubomir, Stippel, Dirk, Bruns, Christiane, Schlitt, Hans, Salame, Ephrem, Kalicinski, Piotr, Acarli, Koray, van Hooff, Maria C., de Veer, Rozanne C., Karam, Vincent, Adam, Rene, Taimr, Pavel, Polak, Wojciech G., Pashtoun, Hasina, Murad, Sarwa Darwish, Corpechot, Christophe, Oniscu, Gabriel C., Metselaar, Herold J., den Hoed, Caroline M., and van der Meer, Adriaan J.

Published
2024
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2. Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Author

Miethke, Alexander G, Moukarzel, Adib, Porta, Gilda, Covarrubias Esquer, Joshue, Czubkowski, Piotr, Ordonez, Felipe, Mosca, Antonella, Aqul, Amal A, Squires, Robert H, Sokal, Etienne, D'Agostino, Daniel, Baumann, Ulrich, D'Antiga, Lorenzo, Kasi, Nagraj, Laborde, Nolwenn, Arikan, Cigdem, Lin, Chuan-Hao, Gilmour, Susan, Mittal, Naveen, Chiou, Fang Kuan, Horslen, Simon P, Huber, Wolf-Dietrich, Jaecklin, Thomas, Nunes, Tiago, Lascau, Anamaria, Longpre, Lara, Mogul, Douglas B, Garner, Will, Vig, Pamela, Hupertz, Vera F, Gonzalez-Peralta, Regino P, Ekong, Udeme, Hartley, Jane, Laverdure, Noemie, Ovchinsky, Nadia, and Thompson, Richard J

Published
2024
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3. Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial

Author

Ovchinsky, Nadia, Aumar, Madeleine, Baker, Alastair, Baumann, Ulrich, Bufler, Philip, Cananzi, Mara, Czubkowski, Piotr, Durmaz, Özlem, Fischer, Ryan, Indolfi, Giuseppe, Karnsakul, Wikrom W, Lacaille, Florence, Lee, Way S, Maggiore, Giuseppe, Rosenthal, Philip, Ruiz, Mathias, Sokal, Etienne, Sturm, Ekkehard, van der Woerd, Wendy, Verkade, Henkjan J, Wehrman, Andrew, Clemson, Christine, Yu, Qifeng, Ni, Quanhong, Ruvido, Jessica, Manganaro, Susan, and Mattsson, Jan P

Published
2024
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5. Familial hypercholesterolaemia in children and adolescents from 48 countries: a cross-sectional study

Author

Dharmayat, Kanika Inamdar, Vallejo-Vaz, Antonio J., Stevens, Christophe A.T., Brandts, Julia M., Lyons, Alexander R.M., Groselj, Urh, Abifadel, Marianne, Aguilar-Salinas, Carlos A., Alhabib, Khalid, Alkhnifsawi, Mutaz, Almahmeed, Wael, Alnouri, Fahad, Alonso, Rodrigo, Al-Rasadi, Khalid, Ashavaid, Tester F., Banach, Maciej, Béliard, Sophie, Binder, Christoph, Bourbon, Mafalda, Chlebus, Krzysztof, Corral, Pablo, Cruz, Diogo, Descamps, Olivier S., Drogari, Euridiki, Durst, Ronen, Ezhov, Marat V., Genest, Jacques, Harada-Shiba, Mariko, Holven, Kirsten B., Humphries, Steve E., Khovidhunkit, Weerapan, Lalic, Katarina, Laufs, Ulrich, Liberopoulos, Evangelos, Roeters van Lennep, Jeanine, Lima-Martinez, Marcos Miguel, Lin, Jie, Maher, Vincent, März, Winfried, Miserez, André R., Mitchenko, Olena, Nawawi, Hapizah, Panayiotou, Andrie G., Paragh, György, Postadzhiyan, Arman, Reda, Ashraf, Reiner, Željko, Reyes, Ximena, Sadiq, Fouzia, Sahebkar, Amirhossein, Schunkert, Heribert, Shek, Aleksandr B., Stroes, Eric, Su, Ta-Chen, Subramaniam, Tavintharan, Susekov, Andrey, Vázquez Cárdenas, Alejandra, Huong Truong, Thanh, Tselepis, Alexandros D., Vohnout, Branislav, Wang, Luya, Yamashita, Shizuya, Al-Sarraf, Ahmad, Al-Sayed, Nasreen, Davletov, Kairat, Dwiputra, Bambang, Gaita, Dan, Kayikcioglu, Meral, Latkovskis, Gustavs, Marais, A. David, Thushara Matthias, Anne, Mirrakhimov, Erkin, Nordestgaard, Børge G., Petrulioniene, Zaneta, Pojskic, Belma, Sadoh, Wilson, Tilney, Myra, Tomlinson, Brian, Tybjærg-Hansen, Anne, Viigimaa, Margus, Catapano, Alberico L., Freiberger, Tomas, Hovingh, G. Kees, Mata, Pedro, Soran, Handrean, Raal, Frederick, Watts, Gerald F., Schreier, Laura, Bañares, Virginia, Greber-Platzer, Susanne, Baumgartner-Kaut, Margot, de Gier, Charlotte, Dieplinger, Hans, Höllerl, Florian, Innerhofer, Reinhold, Karall, Daniela, Lischka, Julia, Ludvik, Bernhard, Mäser, Martin, Scholl-Bürgi, Sabine, Thajer, Alexandra, Toplak, Hermann, Demeure, Fabian, Mertens, Ann, Balligand, Jean-Luc, Stephenne, Xavier, Sokal, Etienne, Petrov, Ivo, Goudev, Assen, Nikolov, Fedya, Tisheva, Snejana, Yotov, Yoto, Tzvetkov, Ivajlo, Hegele, Robert A, Gaudet, Daniel, Brunham, Liam, Ruel, Isabelle, McCrindle, Brian, Cuevas, Ada, Perica, Dražen, Symeonides, Phivos, Trogkanis, Efstratios, Kostis, Andreas, Ioannou, Andreas, Mouzarou, Angeliki, Georgiou, Anthoula, Stylianou, Andreas, Miltiadous, George, Iacovides, Paris, Deltas, Constantinos, Vrablik, Michal, Urbanova, Zuzana, Jesina, Pavel, Tichy, Lukas, Hyanek, Josef, Dvorakova, Jana, Cepova, Jana, Sykora, Josef, Buresova, Kristyna, Pipek, Michal, Pistkova, Eva, Bartkova, Ivana, S|ulakova, Astrid, Toukalkova, Lenka, Spenerova, Michaela, Maly, Jan, Benn, Marianne, Bendary, Ahmed, Elbahry, Atef, Ferrières, Jean, Ferrieres, Dorota, Peretti, Noel, Bruckert, Eric, Gallo, Antonio, Valero, René, Mourre, Florian, Aouchiche, Karine, Reynaud, Rachel, Tounian, Patrick, Lemale, Julie, Boccara, Franck, Moulin, Philippe, Charrières, Sybil, Di Filippo, Mathilde, Cariou, Bertrand, Paillard, François, Dourmap, Caroline, Pradignac, Alain, Verges, Bruno, Simoneau, Isabelle, Farnier, Michel, Cottin, Yves, Yelnik, Cecile, Hankard, Regis, Schiele, François, Durlach, Vincent, Sultan, Ariane, Carrié, Alain, Rabès, Jean-Pierre, Sanin, Veronika, Schmieder, Raphael S., Ates, Sara, Rizos, Christos V., Skoumas, Ioannis, Tziomalos, Konstantinos, Rallidis, Loukianos, Kotsis, Vasileios, Doumas, Michalis, Skalidis, Emmanouil, Kolovou, Genovefa, Kolovou, Vana, Garoufi, Anastasia, Koutagiar, Iosif, Polychronopoulos, Georgios, Kiouri, Estela, Antza, Christina, Zacharis, Evangelos, Attilakos, Achilleas, Sfikas, George, Koumaras, Charalambos, Anagnostis, Panagiotis, Anastasiou, Georgia, Liamis, George, Adamidis, Petros-Spyridon, Milionis, Haralambos, Lambadiari, Vaia, Stabouli, Stella, Filippatos, Theodosios, Mollaki, Vicky, Tsaroumi, Anastasia, Lamari, Frida, Proyias, Pavlos, Harangi, Mariann, Reddy, Lakshmi Lavanya, Shah, Swarup A. V, Ponde, Chandrashekhar K., Dalal, Jamshed J., Sawhney, Jitendra P.S., Verma, Ishwar C., Hosseini, Susan, Jamialahmadi, Tannaz, Alareedh, Mohammed, Shaghee, Foaad, Rhadi, Sabah Hasan, Abduljalal, Maryam, Alfil, Sarmad, Kareem, Huda, Cohen, Hofit, Leitersdorf, Eran, Schurr, Daniel, Shpitzen, Shoshi, Arca, Marcello, Averna, Maurizio, Bertolini, Stefano, Calandra, Sebastiano, Tarugi, Patrizia, Casula, Manuela, Galimberti, Federica, Gazzotti, Marta, Olmastroni, Elena, Sarzani, Riccardo, Ferri, Claudio, Repetti, Elena, Giorgino, Francesco, Suppressa, Patrizia, Bossi, Antonio Carlo, Borghi, Claudio, Muntoni, Sandro, Cipollone, Francesco, Scicali, Roberto, Pujia, Arturo, Passaro, Angelina, Berteotti, Martina, Pecchioli, Valerio, Pisciotta, Livia, Mandraffino, Giuseppe, Pellegatta, Fabio, Mombelli, Giuliana, Branchi, Adriana, Fiorenza, Anna Maria, Pederiva, Cristina, Werba, José Pablo, Parati, Gianfranco, Nascimbeni, Fabio, Iughetti, Lorenzo, Fortunato, Giuliana, Cavallaro, Raimondo, Iannuzzo, Gabriella, Calabrò, Paolo, Cefalù, Angelo Baldassare, Capra, Maria Elena, Zambon, Alberto, Pirro, Matteo, Sbrana, Francesco, Trenti, Chiara, Minicocci, Ilenia, Federici, Massimo, Del Ben, Maria, Buonuomo, Paola Sabrina, Moffa, Simona, Pipolo, Antonio, Citroni, Nadia, Guardamagna, Ornella, Lia, Salvatore, Benso, Andrea, Biolo, Gianni Biolo, Maroni, Lorenzo, Lupi, Alessandro, Bonanni, Luca, Rinaldi, Elisabetta, Zenti, Maria Grazia, Masuda, Daisaku, Mahfouz, Linda, Jambart, Selim, Ayoub, Carine, Ghaleb, Youmna, Kasim, Noor Alicezah Mohd, Nor, Noor Shafina Mohd, Al-Khateeb, Alyaa, Kadir, Siti Hamimah Sheikh Abdul, Chua, Yung-An, Razman, Aimi Zafira, Nazli, Sukma Azureen, Ranai, Norashikin Mohd, Latif, Ahmad Zubaidi Abd, Torres, María Teresa Magaña, Mehta, Roopa, Martagon, Alexandro J., Ramirez, Gabriela A. Galan, Antonio-Villa, Neftali Eduardo, Vargas-Vazquez, Arsenio, Elias-Lopez, Daniel, Retana, Gustavo Gonzalez, Encinas, Bethsabel Rodrıguez, Macıas, Jose J. Ceballos, Zazueta, Alejandro Romero, Alvarado, Rocio Martinez, Portano, Julieta D. Morales, Lopez, Humberto Alvares, Sauque-Reyna, Leobardo, Gomez Herrera, Laura G., Simental Mendia, Luis E., Aguilar, Humberto Garcia, Cooremans, Elizabeth Ramirez, Aparicio, Berenice Pe~na, Zubieta, Victoria Mendoza, Gonzalez, Perla A. Carrillo, Ferreira-Hermosillo, Aldo, Portilla, Nacu Caracas, Dominguez, Guadalupe Jimenez, Garcia, Alinna Y. Ruiz, Arriaga Cazares, Hector E., Gonzalez Gonzalez, Jesus R., Mendez Valencia, Carla V., Padilla Padilla, Francisco G., Prado, Ramon Madriz, De los Rios Ibarra, Manuel O., Arjona Villica~na, Ruy D., Acevedo Rivera, Karina J., Carrera, Ricardo Allende, Alvarez, Jose A., Amezcua Martinez, Jose C., Barrera Bustillo, Manuel de los Reyes, Vargas, Gonzalo Carazo, Chacon, Roberto Contreras, Figueroa Andrade, Mario H., Ortega, Ashanty Flores, Alcala, Hector Garcia, Garcia de Leon, Laura E., Guzman, Berenice Garcia, Gardu~no Garcia, Jose J., Garnica Cuellar, Juan C., Gomez Cruz, Jose R., Garcia, Anell Hernandez, Holguin Almada, Jesus R., Herrera, Ursulo Juarez, Sobrevilla, Fabiola Lugo, Rodriguez, Eduardo Marquez, Sibaja, Cristina Martinez, Medrano Rodriguez, Alma B., Morales Oyervides, Jose C., Perez Vazquez, Daniel I., Reyes Rodriguez, Eduardo A., Osorio, Ma. Ludivina Robles, Saucedo, Juan Rosas, Tamayo, Margarita Torres, Valdez Talavera, Luis A., Vera Arroyo, Luis E., Zepeda Carrillo, Eloy A., Galema-Boers, Annette, Weigman, Albert, Bogsrud, Martin P., Malik, Munir, Shah, Saeedullah, Khan, Sabeen Abid, Rana, Muhammad Asim, Batool, Hijab, Starostecka, Ewa, Konopka, Agnieszka, Lewek, Joanna, Bielecka-Dąbrowa, Agata, Gach, Agnieszka, Jóźwiak, Jacek, Pajkowski, Marcin, Romanowska-Kocejko, Marzena, Żarczyńska-Buchowiecka, Marta, Hellmann, Marcin, Chmara, Magdalena, Wasąg, Bartosz, Parczewska, Aleksandra, Gilis-Malinowska, Natasza, Borowiec-Wolna, Justyna, Stróżyk, Aneta, Michalska-Grzonkowska, Aleksandra, Chlebus, Izabela, Kleinschmidt, Mariola, Wojtecka, Agnieszka, Zdrojewski, Tomasz, Myśliwiec, Małgorzata, Hennig, Matylda, Medeiros, Ana Margarida, Alves, Ana Catarina, Almeida, Ana Filipa, Lopes, Andreia, Guerra, António, Bilhoto, Carla, Simões, Fernando, Silva, Francisco, Lobarinhas, Goreti, Gama, Guida, Palma, Isabel, Salgado, José Miguel, Matos, Luísa Diogo, Moura, Márcio de, Virtuoso, Maria João, Tavares, Mónica, Ferreira, Patrícia, Pais, Patrícia, Garcia, Paula, Coelho, Raquel, Ribeiro, Raquel, Correia, Susana, Sadykova, Dinara, Slastnikova, Evgenia, Alammari, Dalal, Mawlawi, Horia Ahmed, Alsahari, Atif, Khudary, Alia Abdullah, Alrowaily, Nawal Lafi, Rajkovic, Natasa, Popovic, Ljiljana, Singh, Sandra, Rasulic, Iva, Petakov, Ana, Lalic, Nebojsa M., Peng, Fabian Kok, Vasanwala, Rashida Farhan, Venkatesh, Sreedharan Aravind, Raslova, Katarina, Fabryova, Lubomira, Nociar, Jan, Šaligova, Jana, Potočňáková, Ludmila, Kozárová, Miriam, Varga, Tibor, Kadurova, Michaela, Debreova, Marianna, Novodvorsky, Peter, Gonova, Katarina, Klabnik, Alexander, Buganova, Ingrid, Battelino, Tadej, Bizjan, Barbara Jenko, Debeljak, Marusa, Kovac, Jernej, Mlinaric, Matej, Molk, Neza, Sikonja, Jaka, Sustar, Ursa, Podkrajsek, Katarina Trebusak, Muñiz-Grijalvo, Ovidio, Díaz-Díaz, Jose Luis, de Andrés, Raimundo, Fuentes-Jiménez, Francisco, Blom, Dirk, Miserez, Eleonore B., Shipton, Janine L., Ganokroj, Poranee, Futema, Marta, Ramaswami, Uma, Alieva, Rano B., Fozilov, Khurshid G., Khoshimov, Shavkat U., Nizamov, Ulugbek I., Abdullaeva, Guzal J., Kan, Liliya E., Abdullaev, Alisher A., Zakirova, Daria V., Do, Doan-Loi, Nguyen, Mai-Ngoc-Thi, Kim, Ngoc-Thanh, Le, Thanh-Tung, Le, Hong-An, Santos, Raul, and Ray, Kausik K.

Published
2024
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7. Interim results from an ongoing, open-label, single-arm trial of odevixibat in progressive familial intrahepatic cholestasis

Author

Thompson, Richard J., Artan, Reha, Baumann, Ulrich, Calvo, Pier Luigi, Czubkowski, Piotr, Dalgic, Buket, D’Antiga, Lorenzo, Di Giorgio, Angelo, Durmaz, Özlem, Gonzalès, Emmanuel, Grammatikopoulos, Tassos, Gupte, Girish, Hardikar, Winita, Houwen, Roderick H.J., Kamath, Binita M., Karpen, Saul J., Lacaille, Florence, Lachaux, Alain, Lainka, Elke, Loomes, Kathleen M., Mack, Cara L., Mattsson, Jan P., McKiernan, Patrick, Ni, Quanhong, Özen, Hasan, Rajwal, Sanjay R., Roquelaure, Bertrand, Shteyer, Eyal, Sokal, Etienne, Sokol, Ronald J., Soufi, Nisreen, Sturm, Ekkehard, Tessier, Mary Elizabeth, van der Woerd, Wendy L., Verkade, Henkjan J., Vittorio, Jennifer M., Wallefors, Terese, Warholic, Natalie, Yu, Qifeng, Horn, Patrick, and Kjems, Lise

Published
2023
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8. Sofosbuvir and Ribavirin Therapy for Children Aged 3 to <12 Years With Hepatitis C Virus Genotype 2 or 3 Infection

Author

Rosenthal, Philip, Schwarz, Kathleen B, Gonzalez‐Peralta, Regino P, Lin, Chuan‐Hao, Kelly, Deidre A, Nightingale, Scott, Balistreri, William F, Bansal, Sanjay, Jonas, Maureen M, Massetto, Benedetta, Brainard, Diana M, Hsueh, Chia‐Hsiang, Shao, Jiang, Parhy, Bandita, Davison, Suzanne, Feiterna‐Sperling, Cornelia, Gillis, Lynette A, Indolfi, Giuseppe, Sokal, Etienne M, Murray, Karen F, and Wirth, Stefan

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Hepatitis - C, Hepatitis, Liver Disease, Clinical Trials and Supportive Activities, Infectious Diseases, Chronic Liver Disease and Cirrhosis, Clinical Research, Emerging Infectious Diseases, Pediatric, Digestive Diseases, Management of diseases and conditions, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, 7.1 Individual care needs, Infection, Good Health and Well Being, Antiviral Agents, Child, Child, Preschool, Drug Combinations, Female, Genotype, Hepacivirus, Hepatitis C, Chronic, Humans, Male, Ribavirin, Sofosbuvir, Sustained Virologic Response, Treatment Outcome, Medical Biochemistry and Metabolomics, Immunology, Gastroenterology & Hepatology, Clinical sciences
Abstract

Currently, the only approved hepatitis C virus (HCV) treatment for children aged

Published
2020

9. Genotype-phenotype relationships of truncating mutations, p.E297G and p.D482G in bile salt export pump deficiency

Author

Felzen, Antonia, van Wessel, Daan B.E., Gonzales, Emmanuel, Thompson, Richard J., Jankowska, Irena, Shneider, Benjamin L., Sokal, Etienne, Grammatikopoulos, Tassos, Kadaristiana, Agustina, Jacquemin, Emmanuel, Spraul, Anne, Lipiński, Patryk, Czubkowski, Piotr, Rock, Nathalie, Shagrani, Mohammad, Broering, Dieter, Nicastro, Emanuele, Kelly, Deirdre, Nebbia, Gabriella, Arnell, Henrik, Fischler, Björn, Hulscher, Jan B.F., Serranti, Daniele, Arikan, Cigdem, Polat, Esra, Debray, Dominique, Lacaille, Florence, Goncalves, Cristina, Hierro, Loreto, Muñoz Bartolo, Gema, Mozer-Glassberg, Yael, Azaz, Amer, Brecelj, Jernej, Dezsőfi, Antal, Calvo, Pier Luigi, Grabhorn, Enke, Hartleif, Steffen, van der Woerd, Wendy J., Kamath, Binita M., Wang, Jian-She, Li, Liting, Durmaz, Özlem, Kerkar, Nanda, Jørgensen, Marianne Hørby, Fischer, Ryan, Jimenez-Rivera, Carolina, Alam, Seema, Cananzi, Mara, Laverdure, Noemie, Ferreira, Cristina Targa, Guerrero, Felipe Ordoñez, Wang, Heng, Sency, Valerie, Kim, Kyung Mo, Chen, Huey-Ling, de Carvalho, Elisa, Fabre, Alexandre, Bernabeu, Jesus Quintero, Zellos, Aglaia, Alonso, Estella M., Sokol, Ronald J., Suchy, Frederick J., Loomes, Kathleen M., McKiernan, Patrick J., Rosenthal, Philip, Turmelle, Yumirle, Horslen, Simon, Schwarz, Kathleen, Bezerra, Jorge A., Wang, Kasper, Hansen, Bettina E., and Verkade, Henkjan J.

Published
2023
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11. THU-092 Enrolling patients in ACLF trials-the need to increase ACLF awareness (DHELIVER trial)

Author

Thabut, Dominique, primary, Rudler, Marika, additional, Janczewska, Ewa, additional, van der Merwe, Schalk, additional, Nevens, Frederik, additional, Lannes, Adrien, additional, Lasser, Luc, additional, Reiberger, Thomas, additional, Brjalin, Vadim, additional, Pavlova, Desislava, additional, Selcanova, Svetlana Adamcova, additional, Katzarov, Krum, additional, Draganov, Ventseslav, additional, Collienne, Christine, additional, Bureau, Christophe, additional, Saviano, Antonio, additional, Toniutto, Pierluigi, additional, González, Javier Martínez, additional, Sánchez, Jordi, additional, Borissova, Julia, additional, Genov, Jordan, additional, Nikolov, Ivaylo, additional, Gustot, Thierry, additional, Pageaux, Georges-Philippe, additional, Antonini, Teresa, additional, Borentain, Patrick, additional, Leroy, Vincent, additional, Perricone, Giovanni, additional, Grønbæk, Henning, additional, Maasoumy, Benjamin, additional, Bruns, Tony, additional, Pukitis, Aldis, additional, Kupčinskas, Juozas, additional, Stardelova, Kalina Grivcheva, additional, Dimitrova, Magdalena Genadieva, additional, Todorovska, Beti, additional, Eggermont, Fabienne, additional, Vainilovich, Yelena, additional, Lin, Frederic, additional, Gordillo, Noelia, additional, Goddemaer, Griet, additional, Najimi, Mustapha, additional, and Sokal, Etienne, additional

Published
2024
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12. Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders—A successful strategy for clinical research of rare diseases

Author

Posset, Roland, Garbade, Sven F, Boy, Nikolas, Burlina, Alberto B, Dionisi‐Vici, Carlo, Dobbelaere, Dries, Garcia‐Cazorla, Angeles, de Lonlay, Pascale, Teles, Elisa Leão, Vara, Roshni, Mew, Nicholas Ah, Batshaw, Mark L, Baumgartner, Matthias R, McCandless, Shawn E, Seminara, Jennifer, Summar, Marshall, Hoffmann, Georg F, Kölker, Stefan, Burgard, Peter, Berry, Susan A, Burrage, Lindsay, Coughlin, Curtis, Diaz, George A, Gallagher, Renata C, Gropman, Andrea, Harding, Cary O, Lee, Brendan, Le Mons, Cynthia, Lawrence Merritt, J, Nagamani, Sandesh CS, Schulze, Andreas, Stricker, Tamar, Tuchman, Mendel, Waisbren, Susan, WeisfeldAdams, James, Wong, Derek, Yudkoff, Marc, Arnoux, JeanBaptiste, Bari&cacute;, Ivo, Bosch, Annet M, Chabrol, Brigitte, Chakrapani, Anupam, CortèsSaladefont, Elisenda, Couce, Maria L, Eyskens, Francois, Laet, Corine, Meirleir, Linda, Freisinger, Peter, Gleich, Florian, Grünewald, Stephanie, Häberle, Johannes, Hwu, WuhLiang, Jalan, Anil, Karall, Daniela, Lindner, Martin, Lund, Allan M, Martinelli, Diego, Murphy, Elaine, Mühlhausen, Chris, Olivieri, Giorgia, Ottolenghi, Chris, Rodrigues, Esmeralda, Rubert, Laura, Sarajlija, Adrijan, Schiff, Manuel, Sokal, Etienne, SykutCegielska, Jolanta, Walter, John H, Williams, Monique, and Zeman, Jiri

Subjects
Clinical Research, Pediatric, Digestive Diseases, Neurodegenerative, 4.2 Evaluation of markers and technologies, Detection, screening and diagnosis, Good Health and Well Being, Cohort Studies, Data Analysis, Delayed Diagnosis, Europe, Female, Humans, Infant, Newborn, Male, Neonatal Screening, North America, Ornithine Carbamoyltransferase Deficiency Disease, Rare Diseases, Urea, Urea Cycle Disorders, Inborn, Urea cycle Disorders, international registry and database, diagnostic methods, Additional individual contributors of the UCDC and the E-IMD consortium, Clinical Sciences, Genetics & Heredity
Abstract

BACKGROUND:To improve our understanding of urea cycle disorders (UCDs) prospectively followed by two North American (NA) and European (EU) patient cohorts. AIMS:Description of the NA and EU patient samples and investigation of the prospects of combined and comparative analyses for individuals with UCDs. METHODS:Retrieval and comparison of the data from 1095 individuals (NA: 620, EU: 475) from two electronic databases. RESULTS:The proportion of females with ornithine transcarbamylase deficiency (fOTC-D), particularly those being asymptomatic (asfOTC-D), was higher in the NA than in the EU sample. Exclusion of asfOTC-D resulted in similar distributions in both samples. The mean age at first symptoms was higher in NA than in EU patients with late onset (LO), but similar for those with early (≤ 28 days) onset (EO) of symptoms. Also, the mean age at diagnosis and diagnostic delay for EO and LO patients were similar in the NA and EU cohorts. In most patients (including fOTC-D), diagnosis was made after the onset of symptoms (59.9%) or by high-risk family screening (24.7%), and less often by newborn screening (8.9%) and prenatal testing (3.7%). Analysis of clinical phenotypes revealed that EO patients presented with more symptoms than LO individuals, but that numbers of symptoms correlated with plasma ammonium concentrations in EO patients only. Liver transplantation was reported for 90 NA and 25 EU patients. CONCLUSIONS:Combined analysis of databases drawn from distinct populations opens the possibility to increase sample sizes for natural history questions, while comparative analysis utilizing differences in approach to treatment can evaluate therapeutic options and enhance long-term outcome studies.

Published
2019

13. Bacteriophage-antibiotic combination therapy against extensively drug-resistant Pseudomonas aeruginosa infection to allow liver transplantation in a toddler

Author

Van Nieuwenhuyse, Brieuc, Van der Linden, Dimitri, Chatzis, Olga, Lood, Cédric, Wagemans, Jeroen, Lavigne, Rob, Schroven, Kaat, Paeshuyse, Jan, de Magnée, Catherine, Sokal, Etienne, Stéphenne, Xavier, Scheers, Isabelle, Rodriguez-Villalobos, Hector, Djebara, Sarah, Merabishvili, Maya, Soentjens, Patrick, and Pirnay, Jean-Paul

Published
2022
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14. Failure to Rescue Pediatric Recipients of Living Donor Liver Transplantation: A Single‐Center Study of Technical Complications in 500 Primary Grafts.

Author

Channaoui, Aniss, de Magnée, Catherine, Tambucci, Roberto, Bonaccorsi‐Riani, Eliano, Pirotte, Thierry, Magasich‐Airola, Natalia, Detaille, Thierry, Houtekie, Laurent, Menten, Renaud, Dumitriu, Dana, van den Hove, Marguerite, Baldin, Pamela, Smets, Françoise, Scheers, Isabelle, Jannone, Giulia, Sokal, Etienne, Stephenne, Xavier, and Reding, Raymond

Abstract

Background: The concept of failure to rescue (FTR) has been used to evaluate the quality of care in several surgical specialties but has not been well‐studied after living donor liver transplantation (LDLT) in children. Methods: This study retrospectively reviewed 500 pediatric LDLT performed at a single center between 1993 and 2022. The recipient outcomes were assessed by means of patient and graft survival rates, retransplantation rates, and arterial/portal/biliary complication rates. Graft and patient losses secondary to these complications were calculated regarding FTR for patients (FTRp) and grafts (FTRg). Results: Overall 1‐ and 5‐year patient survival rates were 94.5% and 92.1%, respectively, the corresponding figures for graft survival being 92.7% and 89.8%. One‐year hepatic artery complication rate was 3.6% (n = 18 cases), the respective rates for portal vein complications and biliary complications being 5.7% (n = 57) and 15.6% (n = 101). One‐year FTRp rates for hepatic artery thrombosis, portal vein thrombosis, anastomotic biliary stricture, and intrahepatic biliary stricture were 28.6%, 9.4%, 3.6%, and 0%, respectively. The corresponding FTRg rates being 21.4%, 6.3%, 0%, and 36.4%. Conclusion: Such novel analytical method may offer valuable insights for optimizing quality of care in pediatric LDLT. [ABSTRACT FROM AUTHOR]

Published
2024
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15. Favorable Outcome after Liver Transplantation in an Infant with Liver Failure Due to Deoxyguanosine Kinase Deficiency.

Author

Grama, Alina, Benţa, Gabriel, Niculae, Alexandru Stefan, Mititelu, Alexandra, Simu, Claudia, Fufezan, Otilia, Stephenne, Xavier, Reding, Raymond, de Magnee, Catherine, Tambucci, Roberto, Sokal, Etienne, and Pop, Tudor Lucian

Subjects
LIVER transplantation, LIVER failure, MITOCHONDRIAL pathology, DEOXYGUANOSINE, MITOCHONDRIAL DNA, RENAL tubular transport disorders
Abstract

Introduction: Deoxyguanosine Kinase (DGUOK) deficiency is a very rare disorder characterized by liver dysfunction, neurological manifestations, and metabolic disorders secondary to severely reduced mitochondrial DNA content. These patients develop early-onset liver failure, and their liver transplantation (LT) indication remains debatable due to the possibility of neurological involvement. Case Report: We present the case of a 6-month-old female diagnosed with DGUOK deficiency who developed liver failure. At 9 months, she underwent a living-related LT with an initial favorable evolution under immunosuppression therapy with tacrolimus. Four months after LT, she presented two prolonged bacterial and Rotavirus enteritis episodes. She developed classical post-transplant complications (severe renal tubular acidosis type IV, secondary to the high tacrolimus level, and post-transplant lymphoproliferative disease) during these episodes. Her condition deteriorated progressively, with reversible hypotonia and significant weight loss. However, the neurological evaluation did not reveal any signs suggestive of the progression of the underlying disease. A few months later, her clinical features and laboratory parameters improved considerably. Conclusions: This case highlights the unpredictable evolution of children with LT for liver failure due to DGUOK deficiency. [ABSTRACT FROM AUTHOR]

Published
2024
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17. Outcomes of surgical management of familial intrahepatic cholestasis 1 and bile salt export protein deficiencies.

Author

Bull, Laura N, Pawlikowska, Ludmila, Strautnieks, Sandra, Jankowska, Irena, Czubkowski, Piotr, Dodge, Jennifer L, Emerick, Karan, Wanty, Catherine, Wali, Sami, Blanchard, Samra, Lacaille, Florence, Byrne, Jane A, van Eerde, Albertien M, Kolho, Kaija-Leena, Houwen, Roderick, Lobritto, Steven, Hupertz, Vera, McClean, Patricia, Mieli-Vergani, Giorgina, Sokal, Etienne, Rosenthal, Philip, Whitington, Peter F, Pawlowska, Joanna, and Thompson, Richard J

Abstract

Progressive familial intrahepatic cholestasis (PFIC) with normal circulating gamma-glutamyl transpeptidase levels can result from mutations in the ATP8B1 gene (encoding familial intrahepatic cholestasis 1 [FIC1] deficiency) or the ABCB11 gene (bile salt export protein [BSEP] deficiency). We investigated the outcomes of partial external biliary diversion, ileal exclusion, and liver transplantation in these two conditions. We conducted a retrospective multicenter study of 42 patients with FIC1 deficiency (FIC1 patients) and 60 patients with BSEP deficiency (BSEP patients) who had undergone one or more surgical procedures (57 diversions, 6 exclusions, and 57 transplants). For surgeries performed prior to transplantation, BSEP patients were divided into two groups, BSEP-common (bearing common missense mutations D482G or E297G, with likely residual function) and BSEP-other. We evaluated clinical and biochemical outcomes in these patients. Overall, diversion improved biochemical parameters, pruritus, and growth, with substantial variation in individual response. BSEP-common or FIC1 patients survived longer after diversion without developing cirrhosis, being listed for or undergoing liver transplantation, or dying, compared to BSEP-other patients. Transplantation resolved cholestasis in all groups. However, FIC1 patients commonly developed hepatic steatosis, diarrhea, and/or pancreatic disease after transplant accompanied by biochemical abnormalities and often had continued poor growth. In BSEP patients with impaired growth, this generally improved after transplantation. Conclusion: Diversion can improve clinical and biochemical status in FIC1 and BSEP deficiencies, but outcomes differ depending on genetic etiology. For many patients, particularly BSEP-other, diversion is not a permanent solution and transplantation is required. Although transplantation resolves cholestasis in patients with FIC1 and BSEP deficiencies, the overall outcome remains unsatisfactory in many FIC1 patients; this is mainly due to extrahepatic manifestations. (Hepatology Communications 2018;2:515-528).

Published
2018

18. Type of calcineurin inhibitor and long-term outcomes following liver transplantation in patients with Primary Biliary Cholangitis- an ELTR study

Author

van Hooff, Maria C., primary, de Veer, Rozanne C., additional, Karam, Vincent, additional, Adam, Rene, additional, Taimr, Pavel, additional, Polak, Wojciech G., additional, Pashtoun, H., additional, Murad, Sarwa Darwish, additional, Corpechot, Christophe, additional, Mirza, Darius, additional, Heneghan, Michael, additional, Lodge, Peter, additional, Oniscu, Gabriel C., additional, Thorburn, Douglas, additional, Allison, Michael, additional, Metselaar, Herold J., additional, den Hoed, Caroline M., additional, van der Meer, Adriaan J., additional, Oniscu, Gabriel, additional, Pratschke, Johann, additional, Manas, Derek, additional, Bennet, William, additional, Line, Pal-Dag, additional, Hoti, Emir, additional, Zieniewicz, Krzysztof, additional, Ericzon, Bo Goran, additional, Fronek, Jiri, additional, Klempnauer, Jurgen L., additional, Rasmussen, Allan, additional, Romagnoli, Renato, additional, Nemec, Petr, additional, Nordin, Arno, additional, Paul, Andreas, additional, De Simone, Paolo, additional, Porte, R.J., additional, Berlakovich, Gabriela, additional, Cherqui, Daniel, additional, Pirenne, Jacques, additional, Sokal, Etienne, additional, Rossi, Giorgio, additional, Candinas, Daniel, additional, Bachellier, Philippe, additional, Rummo, Oleg, additional, Boudjema, Karim, additional, Mrzljak, Anna, additional, Soubrane, Olivier, additional, Metselaar, Herold, additional, Schneeberger, Stefan, additional, Navarro, Francis, additional, Berney, Thierry, additional, Duvoux, Christophe, additional, Colledan, Michele, additional, De Carlis, Luciano, additional, Boillot, Olivier, additional, Hardwigsen, Jean, additional, Pruvot, Francois Rene, additional, Suc, Bertrand, additional, Vivarelli, Marco, additional, Clavien, Pierre Alain, additional, Lang, Hauke, additional, Kosieradzki, Maciej, additional, Berrevoet, Frederik, additional, Heyd, Bruno, additional, Cescon, Matteo, additional, Chiche, Laurence, additional, Kochs, Eberhard, additional, Baccarani, Umberto, additional, Detry, Olivier, additional, Bartels, Michael, additional, Rossi, Massimo, additional, Scatton, Olivier, additional, Papanikolaou, Vasileios, additional, Alwayn, Ian, additional, Schemmer, Peter, additional, Senninger, N., additional, Ducerf, Christian, additional, Di Benedetto, Fabrizio, additional, Tisone, Giuseppe, additional, Nadalin, Silvio, additional, Mathe, Zoltan, additional, Ribnikar, Marija, additional, Settmacher, Utz, additional, Becker, Thomas, additional, Silva, Nuno, additional, Daniel, Jorge, additional, Popescu, Irinel, additional, Lucidi, Valerio, additional, Bechstein, Wolf O., additional, Decaens, Thomas, additional, Gugenheim, Jean, additional, Gruttadauria, Salvatore, additional, Zamboni, Frausto, additional, Zeytunlu, Murat, additional, Kalff, Jorg C., additional, Vali, Toomas, additional, Tokat, Yaman, additional, Klar, Ernst, additional, Janek, Julius, additional, Kilic, Murat, additional, Katzarov, Krum, additional, Fisher, Lutz, additional, Buc, Emmanuel, additional, Castagneto, Marco, additional, Unek, Tarkan, additional, Spassov, Lubomir, additional, Stippel, Dirk, additional, Bruns, Christiane, additional, Schlitt, Hans, additional, Salame, Ephrem, additional, Kalicinski, Piotr, additional, and Acarli, Koray, additional

Published
2024
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19. Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study

Author

Gonzales, Emmanuel, Hardikar, Winita, Stormon, Michael, Baker, Alastair, Hierro, Loreto, Gliwicz, Dorota, Lacaille, Florence, Lachaux, Alain, Sturm, Ekkehard, Setchell, Kenneth D R, Kennedy, Ciara, Dorenbaum, Alejandro, Steinmetz, Jana, Desai, Nirav K, Wardle, Andrew J, Garner, Will, Vig, Pamela, Jaecklin, Thomas, Sokal, Etienne M, and Jacquemin, Emmanuel

Published
2021
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20. A phase II study of human allogeneic liver-derived progenitor cell therapy for acute-on-chronic liver failure and acute decompensation

Author

Nevens, Frederik, Gustot, Thierry, Laterre, Pierre-François, Lasser, Luc L., Haralampiev, Lyudmil E., Vargas, Victor, Lyubomirova, Desislava, Albillos, Agustin, Najimi, Mustapha, Michel, Sébastien, Stoykov, Ivaylo, Gordillo, Noelia, Vainilovich, Yelena, Barthel, Virginie, Clerget-Chossat, Nathalie, and Sokal, Etienne M.

Published
2021
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22. Immune regulation and therapeutic application of T regulatory cells in liver diseases

Author

Ajith, Ananya, Merimi, Makram, Arki, Mandana Kazem, Hossein-khannazer, Nikoo, Najar, Mehdi, Vosough, Massoud, Sokal, Etienne E.M., Najimi, Mustapha, Ajith, Ananya, Merimi, Makram, Arki, Mandana Kazem, Hossein-khannazer, Nikoo, Najar, Mehdi, Vosough, Massoud, Sokal, Etienne E.M., and Najimi, Mustapha

Abstract

CD4+ CD25+ FOXP3+ T regulatory cells (Tregs) are a subset of the immunomodulatory cell population that can inhibit both innate and adaptive immunity by various regulatory mechanisms. In hepatic microenvironment, proliferation, plasticity, migration, and function of Tregs are interrelated to the remaining immune cells and their secreted cytokines and chemokines. In normal conditions, Tregs protect the liver from inflammatory and auto-immune responses, while disruption of this crosstalk between Tregs and other immune cells may result in the progression of chronic liver diseases and the development of hepatic malignancy. In this review, we analyze the deviance of this protective nature of Tregs in response to chronic inflammation and its involvement in inducing liver fibrosis, cirrhosis, and hepatocellular carcinoma. We will also provide a detailed emphasis on the relevance of Tregs as an effective immunotherapeutic option for autoimmune diseases, liver transplantation, and chronic liver diseases including liver cancer., SCOPUS: re.j, info:eu-repo/semantics/published

Published
2024

25. Genotype correlates with the natural history of severe bile salt export pump deficiency

Author

van Wessel, Daan B.E., Thompson, Richard J., Gonzales, Emmanuel, Jankowska, Irena, Sokal, Etienne, Grammatikopoulos, Tassos, Kadaristiana, Agustina, Jacquemin, Emmanuel, Spraul, Anne, Lipiński, Patryk, Czubkowski, Piotr, Rock, Nathalie, Shagrani, Mohammad, Broering, Dieter, Algoufi, Talal, Mazhar, Nejat, Nicastro, Emanuele, Kelly, Deirdre A., Nebbia, Gabriella, Arnell, Henrik, Björn Fischler, Hulscher, Jan B.F., Serranti, Daniele, Arikan, Cigdem, Polat, Esra, Debray, Dominique, Lacaille, Florence, Goncalves, Cristina, Hierro, Loreto, Muñoz Bartolo, Gema, Mozer-Glassberg, Yael, Azaz, Amer, Brecelj, Jernej, Dezsőfi, Antal, Calvo, Pier Luigi, Grabhorn, Enke, Sturm, Ekkehard, van der Woerd, Wendy J., Kamath, Binita M., Wang, Jian-She, Li, Liting, Durmaz, Özlem, Onal, Zerrin, Bunt, Ton M.G., Hansen, Bettina E., and Verkade, Henkjan J.

Published
2020
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29. Familial hypercholesterolaemia in children and adolescents from 48 countries: a cross-sectional study

Author

Dharmayat, Kanika Inamdar, primary, Vallejo-Vaz, Antonio J., additional, Stevens, Christophe A.T., additional, Brandts, Julia M., additional, Lyons, Alexander R.M., additional, Groselj, Urh, additional, Abifadel, Marianne, additional, Aguilar-Salinas, Carlos A., additional, Alhabib, Khalid, additional, Alkhnifsawi, Mutaz, additional, Almahmeed, Wael, additional, Alnouri, Fahad, additional, Alonso, Rodrigo, additional, Al-Rasadi, Khalid, additional, Ashavaid, Tester F., additional, Banach, Maciej, additional, Béliard, Sophie, additional, Binder, Christoph, additional, Bourbon, Mafalda, additional, Chlebus, Krzysztof, additional, Corral, Pablo, additional, Cruz, Diogo, additional, Descamps, Olivier S., additional, Drogari, Euridiki, additional, Durst, Ronen, additional, Ezhov, Marat V., additional, Genest, Jacques, additional, Harada-Shiba, Mariko, additional, Holven, Kirsten B., additional, Humphries, Steve E., additional, Khovidhunkit, Weerapan, additional, Lalic, Katarina, additional, Laufs, Ulrich, additional, Liberopoulos, Evangelos, additional, Roeters van Lennep, Jeanine, additional, Lima-Martinez, Marcos Miguel, additional, Lin, Jie, additional, Maher, Vincent, additional, März, Winfried, additional, Miserez, André R., additional, Mitchenko, Olena, additional, Nawawi, Hapizah, additional, Panayiotou, Andrie G., additional, Paragh, György, additional, Postadzhiyan, Arman, additional, Reda, Ashraf, additional, Reiner, Željko, additional, Reyes, Ximena, additional, Sadiq, Fouzia, additional, Sahebkar, Amirhossein, additional, Schunkert, Heribert, additional, Shek, Aleksandr B., additional, Stroes, Eric, additional, Su, Ta-Chen, additional, Subramaniam, Tavintharan, additional, Susekov, Andrey, additional, Vázquez Cárdenas, Alejandra, additional, Huong Truong, Thanh, additional, Tselepis, Alexandros D., additional, Vohnout, Branislav, additional, Wang, Luya, additional, Yamashita, Shizuya, additional, Al-Sarraf, Ahmad, additional, Al-Sayed, Nasreen, additional, Davletov, Kairat, additional, Dwiputra, Bambang, additional, Gaita, Dan, additional, Kayikcioglu, Meral, additional, Latkovskis, Gustavs, additional, Marais, A. David, additional, Thushara Matthias, Anne, additional, Mirrakhimov, Erkin, additional, Nordestgaard, Børge G., additional, Petrulioniene, Zaneta, additional, Pojskic, Belma, additional, Sadoh, Wilson, additional, Tilney, Myra, additional, Tomlinson, Brian, additional, Tybjærg-Hansen, Anne, additional, Viigimaa, Margus, additional, Catapano, Alberico L., additional, Freiberger, Tomas, additional, Hovingh, G. Kees, additional, Mata, Pedro, additional, Soran, Handrean, additional, Raal, Frederick, additional, Watts, Gerald F., additional, Schreier, Laura, additional, Bañares, Virginia, additional, Greber-Platzer, Susanne, additional, Baumgartner-Kaut, Margot, additional, de Gier, Charlotte, additional, Dieplinger, Hans, additional, Höllerl, Florian, additional, Innerhofer, Reinhold, additional, Karall, Daniela, additional, Lischka, Julia, additional, Ludvik, Bernhard, additional, Mäser, Martin, additional, Scholl-Bürgi, Sabine, additional, Thajer, Alexandra, additional, Toplak, Hermann, additional, Demeure, Fabian, additional, Mertens, Ann, additional, Balligand, Jean-Luc, additional, Stephenne, Xavier, additional, Sokal, Etienne, additional, Petrov, Ivo, additional, Goudev, Assen, additional, Nikolov, Fedya, additional, Tisheva, Snejana, additional, Yotov, Yoto, additional, Tzvetkov, Ivajlo, additional, Hegele, Robert A, additional, Gaudet, Daniel, additional, Brunham, Liam, additional, Ruel, Isabelle, additional, McCrindle, Brian, additional, Cuevas, Ada, additional, Perica, Dražen, additional, Symeonides, Phivos, additional, Trogkanis, Efstratios, additional, Kostis, Andreas, additional, Ioannou, Andreas, additional, Mouzarou, Angeliki, additional, Georgiou, Anthoula, additional, Stylianou, Andreas, additional, Miltiadous, George, additional, Iacovides, Paris, additional, Deltas, Constantinos, additional, Vrablik, Michal, additional, Urbanova, Zuzana, additional, Jesina, Pavel, additional, Tichy, Lukas, additional, Hyanek, Josef, additional, Dvorakova, Jana, additional, Cepova, Jana, additional, Sykora, Josef, additional, Buresova, Kristyna, additional, Pipek, Michal, additional, Pistkova, Eva, additional, Bartkova, Ivana, additional, S|ulakova, Astrid, additional, Toukalkova, Lenka, additional, Spenerova, Michaela, additional, Maly, Jan, additional, Benn, Marianne, additional, Bendary, Ahmed, additional, Elbahry, Atef, additional, Ferrières, Jean, additional, Ferrieres, Dorota, additional, Peretti, Noel, additional, Bruckert, Eric, additional, Gallo, Antonio, additional, Valero, René, additional, Mourre, Florian, additional, Aouchiche, Karine, additional, Reynaud, Rachel, additional, Tounian, Patrick, additional, Lemale, Julie, additional, Boccara, Franck, additional, Moulin, Philippe, additional, Charrières, Sybil, additional, Di Filippo, Mathilde, additional, Cariou, Bertrand, additional, Paillard, François, additional, Dourmap, Caroline, additional, Pradignac, Alain, additional, Verges, Bruno, additional, Simoneau, Isabelle, additional, Farnier, Michel, additional, Cottin, Yves, additional, Yelnik, Cecile, additional, Hankard, Regis, additional, Schiele, François, additional, Durlach, Vincent, additional, Sultan, Ariane, additional, Carrié, Alain, additional, Rabès, Jean-Pierre, additional, Sanin, Veronika, additional, Schmieder, Roland, additional, Ates, Sara, additional, Rizos, Christos V., additional, Skoumas, Ioannis, additional, Tziomalos, Konstantinos, additional, Rallidis, Loukianos, additional, Kotsis, Vasileios, additional, Doumas, Michalis, additional, Skalidis, Emmanouil, additional, Kolovou, Genovefa, additional, Kolovou, Vana, additional, Garoufi, Anastasia, additional, Koutagiar, Iosif, additional, Polychronopoulos, Georgios, additional, Kiouri, Estela, additional, Antza, Christina, additional, Zacharis, Evangelos, additional, Attilakos, Achilleas, additional, Sfikas, George, additional, Koumaras, Charalambos, additional, Anagnostis, Panagiotis, additional, Anastasiou, Georgia, additional, Liamis, George, additional, Adamidis, Petros-Spyridon, additional, Milionis, Haralambos, additional, Lambadiari, Vaia, additional, Stabouli, Stella, additional, Filippatos, Theodosios, additional, Mollaki, Vicky, additional, Tsaroumi, Anastasia, additional, Lamari, Frida, additional, Proyias, Pavlos, additional, Harangi, Mariann, additional, Reddy, Lakshmi Lavanya, additional, Shah, Swarup A. V, additional, Ponde, Chandrashekhar K., additional, Dalal, Jamshed J., additional, Sawhney, Jitendra P.S., additional, Verma, Ishwar C., additional, Hosseini, Susan, additional, Jamialahmadi, Tannaz, additional, Alareedh, Mohammed, additional, Shaghee, Foaad, additional, Rhadi, Sabah Hasan, additional, Abduljalal, Maryam, additional, Alfil, Sarmad, additional, Kareem, Huda, additional, Cohen, Hofit, additional, Leitersdorf, Eran, additional, Schurr, Daniel, additional, Shpitzen, Shoshi, additional, Arca, Marcello, additional, Averna, Maurizio, additional, Bertolini, Stefano, additional, Calandra, Sebastiano, additional, Tarugi, Patrizia, additional, Casula, Manuela, additional, Galimberti, Federica, additional, Gazzotti, Marta, additional, Olmastroni, Elena, additional, Sarzani, Riccardo, additional, Ferri, Claudio, additional, Repetti, Elena, additional, Giorgino, Francesco, additional, Suppressa, Patrizia, additional, Bossi, Antonio Carlo, additional, Borghi, Claudio, additional, Muntoni, Sandro, additional, Cipollone, Francesco, additional, Scicali, Roberto, additional, Pujia, Arturo, additional, Passaro, Angelina, additional, Berteotti, Martina, additional, Pecchioli, Valerio, additional, Pisciotta, Livia, additional, Mandraffino, Giuseppe, additional, Pellegatta, Fabio, additional, Mombelli, Giuliana, additional, Branchi, Adriana, additional, Fiorenza, Anna Maria, additional, Pederiva, Cristina, additional, Werba, José Pablo, additional, Parati, Gianfranco, additional, Nascimbeni, Fabio, additional, Iughetti, Lorenzo, additional, Fortunato, Giuliana, additional, Cavallaro, Raimondo, additional, Iannuzzo, Gabriella, additional, Calabrò, Paolo, additional, Cefalù, Angelo Baldassare, additional, Capra, Maria Elena, additional, Zambon, Alberto, additional, Pirro, Matteo, additional, Sbrana, Francesco, additional, Trenti, Chiara, additional, Minicocci, Ilenia, additional, Federici, Massimo, additional, Del Ben, Maria, additional, Buonuomo, Paola Sabrina, additional, Moffa, Simona, additional, Pipolo, Antonio, additional, Citroni, Nadia, additional, Guardamagna, Ornella, additional, Lia, Salvatore, additional, Benso, Andrea, additional, Biolo, Gianni Biolo, additional, Maroni, Lorenzo, additional, Lupi, Alessandro, additional, Bonanni, Luca, additional, Rinaldi, Elisabetta, additional, Zenti, Maria Grazia, additional, Masuda, Daisaku, additional, Mahfouz, Linda, additional, Jambart, Selim, additional, Ayoub, Carine, additional, Ghaleb, Youmna, additional, Kasim, Noor Alicezah Mohd, additional, Nor, Noor Shafina Mohd, additional, Al-Khateeb, Alyaa, additional, Kadir, Siti Hamimah Sheikh Abdul, additional, Chua, Yung-An, additional, Razman, Aimi Zafira, additional, Nazli, Sukma Azureen, additional, Ranai, Norashikin Mohd, additional, Latif, Ahmad Zubaidi Abd, additional, Torres, María Teresa Magaña, additional, Mehta, Roopa, additional, Martagon, Alexandro J., additional, Ramirez, Gabriela A. Galan, additional, Antonio-Villa, Neftali Eduardo, additional, Vargas-Vazquez, Arsenio, additional, Elias-Lopez, Daniel, additional, Retana, Gustavo Gonzalez, additional, Encinas, Bethsabel Rodriguez, additional, Macias, Jose J. Ceballos, additional, Zazueta, Alejandro Romero, additional, Alvarado, Rocio Martinez, additional, Portano, Julieta D. Morales, additional, Lopez, Humberto Alvares, additional, Sauque-Reyna, Leobardo, additional, Gomez Herrera, Laura G., additional, Simental Mendia, Luis E., additional, Aguilar, Humberto Garcia, additional, Cooremans, Elizabeth Ramirez, additional, Aparicio, Berenice Pe~na, additional, Zubieta, Victoria Mendoza, additional, Gonzalez, Perla A. Carrillo, additional, Ferreira-Hermosillo, Aldo, additional, Portilla, Nacu Caracas, additional, Dominguez, Guadalupe Jimenez, additional, Garcia, Alinna Y. Ruiz, additional, Arriaga Cazares, Hector E., additional, Gonzalez Gonzalez, Jesus R., additional, Mendez Valencia, Carla V., additional, Padilla Padilla, Francisco G., additional, Prado, Ramon Madriz, additional, De los Rios Ibarra, Manuel O., additional, Arjona Villica~na, Ruy D., additional, Acevedo Rivera, Karina J., additional, Carrera, Ricardo Allende, additional, Alvarez, Jose A., additional, Amezcua Martinez, Jose C., additional, Barrera Bustillo, Manuel de los Reyes, additional, Vargas, Gonzalo Carazo, additional, Chacon, Roberto Contreras, additional, Figueroa Andrade, Mario H., additional, Ortega, Ashanty Flores, additional, Alcala, Hector Garcia, additional, Garcia de Leon, Laura E., additional, Guzman, Berenice Garcia, additional, Gardu~no Garcia, Jose J., additional, Garnica Cuellar, Juan C., additional, Gomez Cruz, Jose R., additional, Garcia, Anell Hernandez, additional, Holguin Almada, Jesus R., additional, Herrera, Ursulo Juarez, additional, Sobrevilla, Fabiola Lugo, additional, Rodriguez, Eduardo Marquez, additional, Sibaja, Cristina Martinez, additional, Medrano Rodriguez, Alma B., additional, Morales Oyervides, Jose C., additional, Perez Vazquez, Daniel I., additional, Reyes Rodriguez, Eduardo A., additional, Osorio, Ma. Ludivina Robles, additional, Saucedo, Juan Rosas, additional, Tamayo, Margarita Torres, additional, Valdez Talavera, Luis A., additional, Vera Arroyo, Luis E., additional, Zepeda Carrillo, Eloy A., additional, Galema-Boers, Annette, additional, Weigman, Albert, additional, Bogsrud, Martin P., additional, Malik, Munir, additional, Shah, Saeedullah, additional, Khan, Sabeen Abid, additional, Rana, Muhammad Asim, additional, Batool, Hijab, additional, Starostecka, Ewa, additional, Konopka, Agnieszka, additional, Lewek, Joanna, additional, Bielecka-Dąbrowa, Agata, additional, Gach, Agnieszka, additional, Jóźwiak, Jacek, additional, Pajkowski, Marcin, additional, Romanowska-Kocejko, Marzena, additional, Żarczyńska-Buchowiecka, Marta, additional, Hellman, Marcin, additional, Chmara, Magdalena, additional, Wasąg, Bartosz, additional, Parczewska, Aleksandra, additional, Gilis-Malinowska, Natasza, additional, Borowiec-Wolna, Justyna, additional, Stróżyk, Aneta, additional, Michalska-Grzonkowska, Aleksandra, additional, Chlebus, Izabela, additional, Kleinschmidt, Mariola, additional, Wojtecka, Agnieszka, additional, Zdrojewski, Tomasz, additional, Myśliwiec, Małgorzata, additional, Hennig, Matylda, additional, Medeiros, Ana Margarida, additional, Alves, Ana Catarina, additional, Almeida, Ana Filipa, additional, Lopes, Andreia, additional, Guerra, António, additional, Bilhoto, Carla, additional, Simões, Fernando, additional, Silva, Francisco, additional, Lobarinhas, Goreti, additional, Gama, Guida, additional, Palma, Isabel, additional, Salgado, José Miguel, additional, Matos, Luísa Diogo, additional, Moura, Márcio de, additional, Virtuoso, Maria João, additional, Tavares, Mónica, additional, Ferreira, Patrícia, additional, Pais, Patrícia, additional, Garcia, Paula, additional, Coelho, Raquel, additional, Ribeiro, Raquel, additional, Correia, Susana, additional, Sadykova, Dinara, additional, Slastnikova, Evgenia, additional, Alammari, Dalal, additional, Mawlawi, Horia Ahmed, additional, Alsahari, Atif, additional, Khudary, Alia Abdullah, additional, Alrowaily, Nawal Lafi, additional, Rajkovic, Natasa, additional, Popovic, Ljiljana, additional, Singh, Sandra, additional, Rasulic, Iva, additional, Petakov, Ana, additional, Lalic, Nebojsa M., additional, Peng, Fabian Kok, additional, Vasanwala, Rashida Farhan, additional, Venkatesh, Sreedharan Aravind, additional, Raslova, Katarina, additional, Fabryova, Lubomira, additional, Nociar, Jan, additional, Šaligova, Jana, additional, Potočňáková, Ludmila, additional, Kozárová, Miriam, additional, Varga, Tibor, additional, Kadurova, Michaela, additional, Debreova, Marianna, additional, Novodvorsky, Peter, additional, Gonova, Katarina, additional, Klabnik, Alexander, additional, Buganova, Ingrid, additional, Battelino, Tadej, additional, Bizjan, Barbara Jenko, additional, Debeljak, Marusa, additional, Kovac, Jernej, additional, Mlinaric, Matej, additional, Molk, Neza, additional, Sikonja, Jaka, additional, Sustar, Ursa, additional, Podkrajsek, Katarina Trebusak, additional, Muñiz-Grijalvo, Ovidio, additional, Díaz-Díaz, Jose Luis, additional, de Andrés, Raimundo, additional, Fuentes-Jiménez, Francisco, additional, Blom, Dirk, additional, Miserez, Eleonore B., additional, Shipton, Janine L., additional, Ganokroj, Poranee, additional, Futema, Marta, additional, Ramaswami, Uma, additional, Alieva, Rano B., additional, Fozilov, Khurshid G., additional, Khoshimov, Shavkat U., additional, Nizamov, Ulugbek I., additional, Abdullaeva, Guzal J., additional, Kan, Liliya E., additional, Abdullaev, Alisher A., additional, Zakirova, Daria V., additional, Do, Doan-Loi, additional, Nguyen, Mai-Ngoc-Thi, additional, Kim, Ngoc-Thanh, additional, Le, Thanh-Tung, additional, Le, Hong-An, additional, Santos, Raul, additional, and Ray, Kausik K., additional

Published
2023
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33. Sofosbuvir–velpatasvir in children 3–17 years old with hepatitis C virus infection

Author

Jonas, Maureen M., Romero, Rene, Rosenthal, Philip, Lin, Chuan‐Hao, Verucchi, Gabriella, Wen, Jessica, Balistreri, William F., Whitworth, Suzanne, Bansal, Sanjay, Leung, Daniel H., Narkewicz, Michael R., Gonzalez‐Peralta, Regino P., Mangia, Alessandra, Karnsakul, Wikrom, Rao, Girish S., Shao, Jiang, Jong, Jan, Parhy, Bandita, Osinusi, Anu, Kersey, Kathryn, Murray, Karen F., Sokal, Etienne M., and Schwarz, Kathleen B.

Abstract

The safety and efficacy of sofosbuvir–velpatasvir in children aged 3–17 years with chronic hepatitis C virus (HCV) infection of any genotype were evaluated. In this Phase 2, multicenter, open‐label study, patients received once daily for 12 weeks either sofosbuvir–velpatasvir 400/100 mg tablet (12–17 years), 200/50 mg low dose tablet or oral granules (3–11 years and ≥17 kg), or 150/37.5 mg oral granules (3–5 years and <17 kg). The efficacy endpoint was sustained virologic response 12 weeks after therapy (SVR12). Dose appropriateness was confirmed by intensive pharmacokinetics in each age group. Among 216 patients treated, 76% had HCV genotype 1% and 12% had genotype 3. Rates of SVR12 were 83% (34/41) among 3–5‐year‐olds, 93% (68/73) among 6–11‐year‐olds, and 95% (97/102) among 12–17‐year‐olds. Only two patients experienced virologic failure. The most common adverse events were headache, fatigue, and nausea in 12–17‐year‐olds; vomiting, cough, and headache in 6–11‐year‐olds; and vomiting in 3–5‐year‐olds. Three patients discontinued treatment because of adverse events. Four patients had serious adverse events; all except auditory hallucination (n= 1) were considered unrelated to study drug. Exposures of sofosbuvir, its metabolite GS‐331007, and velpatasvir were comparable to those in adults in prior Phase 2/3 studies. Population pharmacokinetic simulations supported weight‐based dosing for children in this age range. The pangenotypic regimen of sofosbuvir–velpatasvir is highly effective and safe in treating children 3–17 years with chronic HCV infection. For treating chronic hepatitis C virus (HCV) infection, the combination antiviral regimen sofosbuvir–velpatasvir is highly effective in treating adults with any HCV genotype.In children, the safety, efficacy, and pharmacokinetics of sofosbuvir–velpatasvir had not been evaluated. For treating chronic hepatitis C virus (HCV) infection, the combination antiviral regimen sofosbuvir–velpatasvir is highly effective in treating adults with any HCV genotype. In children, the safety, efficacy, and pharmacokinetics of sofosbuvir–velpatasvir had not been evaluated. In 216 children aged 3–17 years with chronic HCV infection, sofosbuvir–velpatasvir given once daily for 12 weeks had a cure rate of 92%.Rates of cure were 95% among 12–17‐year‐olds (97/102), 93% in 6–11‐year‐olds (68/73), and 83% in 3–5‐year‐olds (34/41).Overall, sofosbuvir–velpatasvir was well tolerated in both tablet and granule formulations; 1.4% (3/216) of participants discontinued because of an adverse event. Pharmacokinetic simulations supported weight‐based dosing for children. In 216 children aged 3–17 years with chronic HCV infection, sofosbuvir–velpatasvir given once daily for 12 weeks had a cure rate of 92%. Rates of cure were 95% among 12–17‐year‐olds (97/102), 93% in 6–11‐year‐olds (68/73), and 83% in 3–5‐year‐olds (34/41). Overall, sofosbuvir–velpatasvir was well tolerated in both tablet and granule formulations; 1.4% (3/216) of participants discontinued because of an adverse event. Pharmacokinetic simulations supported weight‐based dosing for children.

Published
2024
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34. ESPGHAN recommendations on treatment of chronic hepatitis C virus infection in adolescents and children including those living in resource‐limited settings.

Author

Indolfi, Giuseppe, Gonzalez‐Peralta, Regino P., Jonas, Maureen M., Sayed, Manal Hamdy‐El, Fischler, Björn, Sokal, Etienne, Wirth, Stefan, Nicastro, Emanuele, Kohlmaier, Benno, Fitzpatrick, Emer, Gonzales, Emmanuel, Junge, Norman, Mancell, Sarah, Mozer‐Glassberg, Yael, Pop, Tudor, Samyn, Marianne, Stephenne, Xavier, and Zellos, Aglaia

Published
2024
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35. P1 Analysis of long-term treatment effects of odevixibat on clinical outcomes in children with progressive familial intrahepatic cholestasis in odevixibat clinical studies vs external controls from the NAPPED database

Author

Hansen, Bettina, primary, Valcheva, Velichka, additional, Yu, Qifeng, additional, Wessel, Daan BE van, additional, Thompson, Richard J, additional, Gonzales, Emmanuel, additional, Jankowska, Irena, additional, Sokal, Etienne, additional, Grammatikopoulos, Tassos, additional, Kadaristiana, Agustina, additional, Jacquemin, Emmanuel, additional, Spraul, Anne, additional, Lipi&nacute;ski, Patryk, additional, Czubkowski, Piotr, additional, Rock, Nathalie, additional, Shagrani, Mohammad, additional, Kumar, Kishwer, additional, Alkuraya, Fowzan, additional, Sateesh, Meddirevula, additional, Broering, Dieter, additional, Nicastro, Emanuele, additional, Kelly, Deirdre A, additional, Nebbia, Gabriella, additional, Arnell, Henrik, additional, Fischler, Björn, additional, Serranti, Daniele, additional, Arikan, Cigdem, additional, Polat, Esra, additional, Debray, Dominique, additional, Lacaille, Florence, additional, Goncalves, Cristina, additional, Hierro, Loreto, additional, Bartolo, Gema Muñoz, additional, Mozer-Glassberg, Yael, additional, Azaz, Amer, additional, Brecelj, Jernej, additional, Dezs&odblac;fi, Antal, additional, Calvo, Pier Luigi, additional, Grabhorn, Enke, additional, Sturm, Ekkehard, additional, Woerd, Wendy L van der, additional, Kamath, Binita M, additional, Wang, Jian-She, additional, Li, Liting, additional, Durmaz, Özlem, additional, Onal, Zerrin, additional, Felzen, Antonia, additional, Nomden, Mark, additional, Sonajalg, Jaak, additional, Stein, Philip, additional, Ni, Quanhong, additional, Clemson, Christine, additional, Mattsson, Jan P, additional, and Verkade, Henkjan J, additional

Published
2023
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40. Evidence for an alternative fatty acid desaturation pathway increasing cancer plasticity

Author

Vriens, Kim, Christen, Stefan, Parik, Sweta, Broekaert, Dorien, Yoshinaga, Kazuaki, Talebi, Ali, Dehairs, Jonas, Escalona-Noguero, Carmen, Schmieder, Roberta, Cornfield, Thomas, Charlton, Catriona, Romero-Pérez, Laura, Rossi, Matteo, Rinaldi, Gianmarco, Orth, Martin F., Boon, Ruben, Kerstens, Axelle, Kwan, Suet Ying, Faubert, Brandon, Méndez-Lucas, Andrés, Kopitz, Charlotte C., Chen, Ting, Fernandez-Garcia, Juan, Duarte, João A. G., Schmitz, Arndt A., Steigemann, Patrick, Najimi, Mustapha, Hägebarth, Andrea, Van Ginderachter, Jo A., Sokal, Etienne, Gotoh, Naohiro, Wong, Kwok-Kin, Verfaillie, Catherine, Derua, Rita, Munck, Sebastian, Yuneva, Mariia, Beretta, Laura, DeBerardinis, Ralph J., Swinnen, Johannes V., Hodson, Leanne, Cassiman, David, Verslype, Chris, Christian, Sven, Grünewald, Sylvia, Grünewald, Thomas G. P., and Fendt, Sarah-Maria

Published
2019
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41. Biological tests carried out on serum/plasma samples from donors of human body material for transplantation: Belgian experience and practical recommendations

Author

Padalko, Elizaveta, Lagrou, Katrien, Delforge, Marie-Luce, Jansens, Hilde, Ectors, Nadine, Pirnay, Jean-Paul, Klykens, Johan, Sokal, Etienne, Muylle, Ludo, Libois, Agnes, Vanderkelen, Alain, Verbeken, Gilbert, Matthys, Conny, Goossens, Dominique, Hanssens, Geert, Baltes, Muriel, and Beele, Hilde

Published
2018
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44. Analysis of safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: data from MARCH-PFIC

Author

Miethke, Alexander, primary, Moukarzel, Adib, additional, Porta, Gilda, additional, Esquer, Joshue Covarrubias, additional, Czubkowski, Piotr, additional, Ordoñez, Felipe, additional, Candusso, Manila, additional, Aqul, Amal A., additional, Squires, Robert H., additional, Sokal, Etienne, additional, D’Agostino, Daniel, additional, Baumann, Ulrich, additional, D’Antiga, Lorenzo, additional, Kasi, Nagraj, additional, Laborde, Nolwenn, additional, Arikan, Cigdem, additional, Lin, Chuan-Hao, additional, Gilmour, Susan, additional, Mittal, Naveen, additional, Chiou, Fang Kuan, additional, Horslen, Simon P., additional, Huber, Wolf-Dietrich, additional, David-Feliciano, Susan, additional, Chien, Elaine, additional, Mogul, Douglas, additional, Garner, Will, additional, Nunes, Tiago, additional, Lascau, Anamaria, additional, Vig, Pamela, additional, Hupertz, Vera, additional, Gonzalez-Peralta, Regino, additional, Ekong, Udeme, additional, Hartley, Jane, additional, Laverdure, Noemie, additional, Ovchinsky, Nadia, additional, and Thompson, Richard, additional

Published
2023
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45. Efficacy and safety outcomes with odevixibat treatment: Pooled data from the phase 3 ASSERT and ASSERT-EXT studies in patients with Alagille syndrome

Author

Ovchinsky, Nadia, primary, Aumar, Madeleine, additional, Baker, Alastair, additional, Baumann, Ulrich, additional, Bufler, Philip, additional, Cananzi, Mara, additional, Durmaz, Ozlem, additional, Fischer, Ryan, additional, Indolfi, Giuseppe, additional, Karnsakul, Wikrom, additional, Lacaille, Florence, additional, Lee, Way Seah, additional, Maggiore, Giuseppe, additional, Rosenthal, Philip, additional, Ruiz, Mathias, additional, Sokal, Etienne, additional, Sturm, Ekkehard, additional, van der Woerd, Wendy L., additional, Verkade, Henkjan J., additional, Wehrman, Andrew, additional, Clemson, Christine, additional, Yu, Qifeng, additional, Ni, Quanhong, additional, Ruvido, Jessica, additional, Manganaro, Susan, additional, Mattsson, Jan, additional, and Czubkowski, Piotr, additional

Published
2023
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46. Safety, tolerability, and preliminary efficacy of ascending doses of Human Allogeneic Liver-derived Progenitor Cells (HepaStem®) in patients with cirrhotic and pre-cirrhotic non-alcoholic steatohepatitis (NASH)

Author

Francque, Sven, primary, Moreno, Christophe, additional, Saliba, Faouzi, additional, Vargas, Victor, additional, Ibañez, Luis, additional, Soriano, German, additional, Genov, Jordan, additional, Katzarov, Krum, additional, Janczewska, Ewa, additional, Iliescu, Elena Laura, additional, Geerts, Anja, additional, Gordillo, Noelia, additional, Vainilovich, Yelena, additional, Najimi, Mustapha, additional, Barthel, Virginie, additional, Lin, Frederic, additional, and Sokal, Etienne, additional

Published
2023
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47. Analysis of long-term treatment effects of odevixibat on clinical outcomes in children with progressive familial intrahepatic cholestasis in odevixibat clinical studies vs external controls from the NAPPED database

Author

Hansen, Bettina, primary, Valcheva, Velichka, additional, Yu, Qifeng, additional, van Wessel, Daan, additional, Thompson, Richard, additional, Gonzalès, Emmanuel, additional, Jankowska, Irena, additional, Sokal, Etienne, additional, Grammatikopoulos, Tassos, additional, Kadaristiana, Agustina, additional, Jacquemin, Emmanuel, additional, Spraul, Anne, additional, Lipiński, Patryk, additional, Czubkowski, Piotr, additional, Rock, Nathalie, additional, Shagrani, Mohammad, additional, Kumar, Kishwer, additional, Alkuraya, Fowzan, additional, Sateesh, Meddirevula, additional, Broering, Dieter Clemens, additional, Nicastro, Emanuele, additional, Kelly, Deirdre, additional, Nebbia, Gabriella, additional, Arnell, Henrik, additional, Fischler, Björn, additional, Serranti, Daniele, additional, Arıkan, Cigdem, additional, Polat, Esra, additional, Debray, Dominique, additional, Lacaille, Florence, additional, Gonçalves, Cristina, additional, Llanillo, Loreto Hierro, additional, Bartolo, Gema Muñoz, additional, Mozer-Glassberg, Yael, additional, Azaz, Amer, additional, Brecelj, Jernej, additional, Dezsőfi, Antal, additional, Calvo, Pier Luigi, additional, Grabhorn, Enke, additional, Sturm, Ekkehard, additional, van der Woerd, Wendy L., additional, Kamath, Binita M., additional, Wang, Jian-She, additional, Liting, Li, additional, Durmaz, Ozlem, additional, Önal, Zerrin, additional, Felzen, Antonia, additional, Nomden, Mark, additional, Sonajalg, Jaak, additional, Stein, Philip, additional, Ni, Quanhong, additional, Clemson, Christine, additional, Mattsson, Jan, additional, and Verkade, Henkjan J., additional

Published
2023
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48. Serum bile acids are associated with native liver survival in patients with Alagille syndrome: results from the GALA study group

Author

Murillo Perez, Carla Fiorella, primary, Vandriel, Shannon M., additional, Wang, Jian-She, additional, Liting, Li, additional, She, Huiyu, additional, Jankowska, Irena, additional, Czubkowski, Piotr, additional, Gliwicz, Dorota, additional, Gonzalès, Emmanuel, additional, Jacquemin, Emmanuel, additional, Bouligand, Jérôme, additional, D’Antiga, Lorenzo, additional, Nicastro, Emanuele, additional, Fischler, Björn, additional, Arnell, Henrik, additional, Siew, Susan, additional, Stormon, Michael, additional, Loomes, Kathleen M., additional, Piccoli, David A., additional, Rand, Elizabeth B., additional, Squires, James E., additional, Karpen, Saul J., additional, Romero, Rene, additional, Kasahara, Mureo, additional, Önal, Zerrin, additional, Sokal, Etienne, additional, Demaret, Tanguy, additional, Wiecek, Sabina, additional, Lacaille, Florence, additional, Debray, Dominique, additional, Hardikar, Winita, additional, Shankar, Sahana, additional, Valentino, Pamela, additional, Sundaram, Shikha, additional, Chaidez, Alexander, additional, Ebel, Noelle, additional, Feinstein, Jeffrey, additional, Mozar-Glazberg, Yael, additional, Lin, Henry, additional, Rock, Nathalie, additional, Verkade, Henkjan J., additional, Jensen, M.K., additional, Jaramillo, Catalina, additional, Kim, Kyungmo, additional, Oh, Seak Hee, additional, Brecelj, Jernej, additional, Alam, Seema, additional, Indolfi, Giuseppe, additional, Blondet, Niviann, additional, Fawaz, Rima, additional, Nastasio, Silvia, additional, Calvo, Pier Luigi, additional, Nebbia, Gabriella, additional, Arıkan, Cigdem, additional, Larson-Nath, Catherine, additional, Zizzo, Andréanne N., additional, Sandahl, Thomas Damgaard, additional, Tzivinikos, Christos, additional, El-Koofy, Nehal, additional, Elmonem, Mohamed, additional, Desai, Dev, additional, Karnsakul, Wikrom, additional, Karthikeyan, Palaniswamy, additional, Bulut, Pinar, additional, Kerkar, Nanda, additional, Wolters, Victorien, additional, Roberts, Amin J, additional, Evans, Helen, additional, Sanchez, Maria Camila, additional, Cavalieri, Maria Lorena, additional, Kelly, Deirdre, additional, Lee, Way Seah, additional, Hajinicolaou, Christina, additional, Lertudomphonwanit, Chatmanee, additional, Fischer, Ryan, additional, Bernabeu, Jesús Quintero, additional, Quiros-Tejeira, Ruben E., additional, Melere, Melina, additional, Carvalho, Elisa, additional, Eshun, John, additional, Zellos, Aglaia, additional, Dezsőfi, Antal, additional, Pinto, Raquel Borges, additional, Schwarz, Kathleen, additional, Rogalidou, Maria, additional, Garcia, Jennifer, additional, Tamara, María Legarda, additional, Beretta, Marisa, additional, Mujawar, Quais, additional, Santos-Silva, Ermelinda, additional, Busoms, Cristina Molera, additional, Lurz, Eberhard, additional, Gonçalves, Cristina, additional, Jimenez-Rivera, Carolina, additional, Bañales, Jesús M., additional, Shah, Uzma, additional, Thompson, Richard, additional, Hansen, Bettina, additional, and Kamath, Binita M., additional

Published
2023
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49. Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): data from the MARCH-ON study

Author

Miethke, Alexander, primary, Moukarzel, Adib, additional, Porta, Gilda, additional, Esquer, Joshue Covarrubias, additional, Czubkowski, Piotr, additional, Ordonez, Felipe, additional, Candusso, Manila, additional, Aqul, Amal A., additional, Squires, Robert H., additional, Sokal, Etienne, additional, D’Agostino, Daniel, additional, Baumann, Ulrich, additional, D’Antiga, Lorenzo, additional, Kasi, Nagraj, additional, Laborde, Nolwenn, additional, Arıkan, Cigdem, additional, Lin, Chuan-Hao, additional, Gilmour, Susan, additional, Mittal, Naveen, additional, Chiou, Fang Kuan, additional, Horslen, Simon P., additional, Huber, Wolf-Dietrich, additional, Nunes, Tiago, additional, Lascau, Anamaria, additional, Longpre, Lara, additional, Mogul, Douglas, additional, Aguilar, Raul, additional, Vig, Pamela, additional, Hupertz, Vera, additional, Gonzalez-Peralta, Regino, additional, Ekong, Udeme, additional, Hartley, Jane, additional, Laverdure, Noemie, additional, Ovchinsky, Nadia, additional, and Thompson, Richard, additional

Published
2023
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50. ACLF course profiles over screening period in the phase IIb DHELIVER study

Author

Nevens, Frederik, primary, Thabut, Dominique, additional, Rudler, Marika, additional, Adrien, Lannes, additional, Lasser, Luc, additional, Brjalin, Vadim, additional, Pageaux, Georges-Philippe, additional, Janczewska, Ewa, additional, Toniutto, Pierluigi, additional, Martinez, Javier, additional, Reiberger, Thomas, additional, Sánchez-Delgado, Jordi, additional, Vargas, Victor, additional, Gustot, Thierry, additional, Maasoumy, Benjamin, additional, Grønbæk, Henning, additional, Bruns, Tony, additional, Bureau, Christophe, additional, Stardelova, Kalina Grivcheva, additional, Pavlova, Desislava, additional, Nikolov, Ivaylo, additional, Katzarov, Krum, additional, Draganov, Ventseslav, additional, Monterde, Vanesa Bernal, additional, Gordillo, Noelia, additional, Vainilovich, Yelena, additional, Najimi, Mustapha, additional, Barthel, Virginie, additional, Lin, Frederic, additional, and Sokal, Etienne, additional

Published
2023
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