학술논문
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial
Document Type
Article
Author
Volpe, Joseph J.; Posner, John; Kellner, Ulrich; Quinlivan, Rosaline; Daron, Aurore; Delstanche, Stéphanie; Bruninx, Romain; Dal Farra, Fabian; Schneider, Olivier; Deconinck, Nicolas; Balikova, Irina; Delbeke, Patricia; Joniau, Inge; Tahon, Valentine; Wittevrongel, Sylvia; De Vos, Elke; Goemans, Nathalie; Casteels, Ingele; De Waele, Liesbeth; Cassiman, Catherine; Prové, Lies; Kinoo, David; Vancampenhout, Lisa; Van Den Hauwe, Marleen; Van Impe, Annelies; Prufer de Queiroz Campos Araujo, Alexandra; Chacon Pereira, Aline; Nardes, Flávia; Haefeli, Lorena; Rossetto, Julia; Ferreira Rebel, Marcos; Almeida Pereira, Jaqueline; Campbell, Craig; Sharan, Sapna; McDonald, Wendy; Scholtes, Cheryl; Mah, Jean; Sframeli, Maria; Chiu, Angela; Hagel, Jane; Oskoui, Maryam; Beneish, Raquel; Cariou-Palmer, Gaela; Pham, Connie; Toffoli, Daniela; Arpin, Stephanie; Turgeon Desilets, Sarah; Wang, Yi; Hu, Chaoping; Huan, Jianfeng; Qian, Chen; Shen, Li; Xiao, Ying; Zhou, Zhenxuan; Li, Hui; Wang, Sujuan; Xiong, Hui; Chang, Xingzhi; Dong, Hui; Liu, Ying; Sang, Tian; Wei, Cuijie; Wen, Jing; Cao, Yiwen; Ly, Xingyao; Zhao, Jingjing; Li, Wenzhu; Qin, Lun; Barisic, Nina; Galiot Delic, Martina; Ivkic, Petra Kristina; Vukojevic, Nenad; Kern, Ivana; Najdanovic, Boris; Skugor, Marin; Servais, Laurent; Boespflug-Tanguy, Odile; Gidaro, Teresa; Seferian, Andreea; De Lucia, Silvana; Barreau, Emmanuel; Mnafek, Nabila; Momtchilova, Marta Milkova; Peche, Helene; Valherie, Carole; Grange, Allison; Lilien, Charlotte; Milascevic, Darko; Tachibana, Shotaro; Ravelli, Claudia; Cardas, Ruxandra; Taytard, Jessica; Aubertin, Guillaume; Vanden Brande, Laure; Davion, Jean-Baptiste; Coopman, Stephanie; Bouacha, Ikram; Debruyne, Philippe; Defoort, Sabine; Derlyn, Gilles; Leroy, Florian; Danjoux, Loïc; Guilbaud, Julie; Desguerre, Isabelle; Barnérias, Christine; Semeraro, Michaela; Bremond-Gignac, Dominique; Bruere, Lenaic; Rateaux, Maxence; Deladrière, Élodie; Germa, Virginie; Pereon, Yann; Mercie, Sandra; Billaud, Fanny; Le Goff, Lucie; Letellier, Guy; Vuillerot, Carole; Portefaix, Aurélie; De-Montferrand, Camille; Le-Goff, Laure; Fontaine, Stephanie; Saidi, Manel; Bouzid, Nabil; Barriere, Aurélie; Tinat, Marie; Kirschner, Janbernd; Dreesbach, Michelle; Lagréze, Wolf; Michaelis, Bettina; Molnar, Fanni; Seger, Dorina; Vogt, Sibylle; Bertini, Enrico; D'Amico, Adele; Petroni, Sergio; Bonetti, Anna Maria; Carlesi, Adelina; Mizzoni, Irene; Bruno, Claudio; Priolo, Enrico; Rao, Giuseppe; Morando, Simone; Tacchetti, Paola; Zuffi, Ambra; Comi, Giacomo Pietro; Brusa, Roberta; Corti, Stefania; Daniele, Velardo; Govoni, Alessandra; Magri, Francesca; Minorini, Valeria; Osnaghi, Silvia Gabriella; Abbati, Francesca; Fassini, Federica; Foa, Michaela; Lopopolo, Amaqlia; Meneri, Megi; Zoppas, Francesca; Parente, Valeria; Baranello, Giovanni; Masson, Riccardo; Bianchi Marzoli, Stefania; Santarsiero, Diletta; Garcia Sierra, Myriam; Tremolada, Gemma; Arnoldi, Maria Teresa; Vigano, Marta; Zanin, Riccardo; Mercuri, Eugenio; Antonaci, Laura; de Sanctis, Roberto; Pane, Marika; Pera, Maria Carmela; Amorelli, Giulia Maria; Barresi, Costanza; D'Amico, Gugliemo; Orazi, Lorenzo; Coratti, Giorgia; Haginoya, Kazuhiro; Kato, Atsuko; Morishita, Yuko; Kira, Ryutaro; Akiyama, Kiyomu; Goto, Miwako; Mori, Yujiro; Okamoto, Misato; Tsutsui, Saki; Takatsuji, Yuta; Tanaka, Aya; Komaki, Hirofumi; Omori, Miina; Suzuki, Ippei; Takeuchi, Mizuki; Todoroki, Daisuke; Watanabe, Seji; Matsubayashi, Tomoko; Inakazu, Emi; Nagura, Hiroe; Suzuki, Akira; Usui, Manami; Ishikawa, Nobutsune; Harada, Yousuke; Fudeyasu, Kenishi; Hirata, Kazuhiko; Michiue, Kana; Ueda, Kazuyuki; Saito, Kayoko; Fujitani, Junko; Arakawa, Reiko; Takano, Kozue; Yashiro, Shigeko; Seki, Maiko; Sano, Nozomi; Fukuyama, Koji; Matsumoto, Yuki; Miyazaki, Hirofumi; Shibata, Minoru; Kobayashi, Kyoko; Nakamura, Yukie; Takeshima, Yasuhiro; Kuma, Moe; Kostera-Pruszczyk, Anna; Fraczek, Anna; Jedrzejowska, Maria; Lusakowska, Anna; Czeszyk-Piotrowicz, Agnieszka; Hautz, Wojciech; Rakusiewicz, Klaudia; Burlewicz, Malgorzata; Gierlak-Wojcicka, Zuzanna; Kepa, Malwina; Sikorski, Adam; Sobieraj, Marcin; Mazurkiewicz-Beldzinska, Maria; Lemska, Anna; Modrzejewska, Sandra; Koberda, Mateusz; Stodolska-Koberda, Urszula; Waskowska, Agnieszka; Kolendo, Jagoda; Sobierajska-Rek, Agnieszka; Steinborn, Barbara; Dalz, Magdalena; Grabowska, Julia; Hajduk, Wojciech; Janasiewicz-Karachitos, Justyna; Klimas, Monika; Stopa, Marcin; Gajewska, Ewa; Pusz, Beata; Vlodavets, Dmitry; Melnik, Evgenia; Leppenen, Natalya; Yupatova, Nataliya; Monakhova, Anastasya; Papina, Yulia; Shidlovsckaia, Olga; Milic Rasic, Vedrana; Brankovic, Vesna; Kosac, Ana; Djokic, Olivera; Jakšic, Vesna; Pepic, Ana; Martinovic, Jelena; Munell Casadesus, Francina; Tizzano, Eduardo; Martín Begué, Nieves; Wolley Dod, Charlotte; Subira, Olaia; Planas Pascual, Bernat; Toro Tamargo, Esther; Madruga Garrido, Marcos; Medina Romero, José David; Salinas, Marta Peña; Nascimento Osorio, Andrés; Díaz Cortés, Ana; Jiménez Gañan, Enrique; Suh, Simone Dowon; Medina Cantillo, Julita; Moya, Obdulia; Padros, Nuria; Urraca, Sandra Roca; Valdivia, Hugo Gonzalez; Pascual Pascual, Samuel; de Manuel, Sofía; Martin, Susana Noval; Burnham, Paul; Espinosa, Sandra; Moreno, Mercedes Martinez; Topaloglu, Haluk; Oncel, Ibrahim; Eroglu Ertugru, Nesibe; Konuskan, Bahadir; Eldem, Bora; Kadayifçilar, Sibel; Alemdaroglu, Ipek; Karaduman, Aynur Ayse; Yilmaz, Oznur Tunca; Bilgin, Neslihan; Sari, Seher; Chiriboga, Claudia; Lee, John J.; Rome-Martin, Donnielle; Day, John W.; Beres, Shannon; Duong, Tina; Gee, Richard; Dunaway Young, Sally; Fuerst-Recktenwald, Sabine; Marquet, Anne; Muelhardt, Nicoletta; Trundell, Dylan; Mazzone, Elena S; Nascimento, Andres; Gerber, Marianne; Gorni, Ksenija; Khwaja, Omar; Kletzl, Heidemarie; Scalco, Renata S; Staunton, Hannah; Yeung, Wai Yin; Martin, Carmen; Fontoura, Paulo; Day, John W
Source
In The Lancet Neurology January 2022 21(1):42-52
Subject
Language
ISSN
1474-4422