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
Mercuri, E.Mazzone, E.S.Deconinck, N.Nascimento, A.Oskoui, M.Saito, K.Vuillerot, C.Baranello, G.Boespflug-Tanguy, O.Servais, L.Goemans, N.Kirschner, J.Kostera-Pruszczyk, A.Gerber, M.Gorni, K.Khwaja, O.Kletzl, H.Scalco, R.S.Fontoura, P.Staunton, H.Yeung, W.Y.Martin, C.Day, J.W.Volpe, J.J.Posner, J.Kellner, U.Quinlivan, R.Fuerst-Recktenwald, S.Marquet, A.Mülhardt, N.Trundell, D.Daron, A.Delstanche, S.Romain, B.Dal Farra, F.Schneider, O.Balikova, I.Delbeke, P.Joniau, I.Tahon, V.Wittevrongel, S.De Vos, E.Casteels, I.De Waele, L.Cassiman, C.Prové, L.Kinoo, D.Vancampenhout, L.Van Den Hauwe, M.Van Impe, A.Prufer de Queiroz Campos Araujo, A.Chacon Pereira, A.Nardes, F.Haefeli, L.Rossetto, J.Almeida Pereira, J.Ferreira Rebel, M.Campbell, C.Sharan, S.McDonald, W.Scholtes, C.Mah, J.Sframeli, M.Chiu, A.Hagel, J.Beneish, R.Pham, C.Toffoli, D.Arpin, S.Turgeon Desilets, S.Wang, Y.Hu, C.Huang, J.Qian, C.Shen, L.Xiao, Y.Zhou, Z.Li, H.Wang, S.Xiong, H.Chang, X.Dong, H.Liu, Y.Sang, T.Wei, C.Wen, J.Cao, Y.Lv, X.Zhao, J.Li, W.Qin, L.Barisic, N.Galiot Delic, M.Ivkic, P.K.Vukojevic, N.Kern, I.Najdanovic, B.Skugor, M.Gidaro, T.Seferian, A.De Lucia, S.Barreau, E.Mnafek, N.Momtchilova, M.M.Peche, H.Valherie, C.Grange, A.Lilien, C.Milascevic, D.Tachibana, S.Ravelli, C.Cardas, R.Vanden Brande, L.Davion, J.-B.Coopman, S.Bouacha, I.Debruyne, P.Defoort, S.Derlyn, G.Leroy, F.Danjoux, L.Guilbaud, J.Desguerre, I.Barnérias, C.Semeraro, M.Bremond-Gignac, D.Bruere, L.Rateaux, M.Deladrière, E.Germa, V.Pereon, Y.Magot, A.Mercie, S.Billaud, F.Le Goff, L.Letellier, G.Portefaix, A.Fontaine, S.De-Montferrand, C.Le-Goff, L.Saidi, M.Bouzid, N.Barriere, A.Tinat, M.Dreesbach, M.Lagréze, W.Michaelis, B.Molnar, F.Seger, D.Vogt, S.Bertini, E.D'Amico, A.Petroni, S.Bonetti, A.M.Carlesi, A.Mizzoni, I.Bruno, C.Priolo, E.Rao, G.Morando, S.Tacchetti, P.Zuffi, A.Comi, G.P.Brusa, R.Corti, S.Daniele, V.Govoni, A.Magri, F.Minorini, V.Osnaghi, S.G.Abbati, F.Fassini, F.Foa, M.Lopopolo, A.Meneri, M.Zoppas, F.Parente, V.Masson, R.Bianchi Marzoli, S.Santarsiero, D.Garcia Sierra, M.Tremolada, G.Arnoldi, M.T.Vigano, M.Zanin, R.Amorelli, G.M.Barresi, C.D'Amico, G.Orazi, L.Coratti, G.Haginoya, K.Kato, A.Morishita, Y.Kira, R.Akiyama, K.Goto, M.Mori, Y.Okamoto, M.Tsutsui, S.Takatsuji, Y.Tanaka, A.Komaki, H.Suzuki, I.Takeuchi, M.Todoroki, D.Watanabe, S.Omori, M.Matsubayashi, T.Inakazu, E.Nagura, H.Suzuki, A.Osaka, H.Ohashi, M.Ishikawa, N.Harada, Y.Fudeyasu, K.Hirata, K.Michiue, K.Ueda, K.Yashiro, S.Seki, M.Sano, N.Uemura, A.Fukuyama, K.Matsumoto, Y.Miyazaki, H.Shibata, M.Kobayashi, K.Nakamura, Y.Takeshima, Y.Kuma, M.Fraczek, A.Jedrzejowska, M.Lusakowska, A.Czeszyk-Piotrowicz, A.Hautz, W.Rakusiewicz, K.Burlewicz, M.Gierlak-Wojcicka, Z.Kepa, M.Sikorski, A.Sobieraj, M.Mazurkiewicz-Beldzinska, M.Lemska, A.Modrzejewska, S.Koberda, M.Stodolska-Koberda, U.Waskowska, A.Kolendo, J.Sobierajska-Rek, A.Steinborn, B.Dalz, M.Grabowska, J.Hajduk, W.Janasiewicz-Karachitos, J.Klimas, M.Stopa, M.Gajewska, E.Pusz, B.Vlodavets, D.Melnik, E.Leppenen, N.Yupatova, N.Monakhova, A.Papina, Y.Shidlovsckaia, O.Milic Rasic, V.Brankovic, V.Kosac, A.Djokic, O.Jakšić, V.Pepic, A.Martinovic, J.Munell Casadesus, F.Tizzano, E.Martín Begué, N.Wolley Dod, C.Subira, O.Planas Pascual, B.Toro Tamargo, E.Madruga Garrido, M.Medina Romero, J.D.Salinas, M.P.Nascimento Osorio, A.Díaz Cortés, A.Jiménez Gañan, E.Suh, S.D.Medina Cantillo, J.Moya, O.Padros, N.Roca Urraca, S.Gonzalez Valdivia, H.Pascual Pascual, S.de Manuel, S.Noval Martin, S.Burnham, P.Espinosa Garcia, S.Martinez Moreno, M.Topaloglu, H.Oncel, I.Eroglu Ertugrul, N.Konuskan, B.Eldem, B.Kadayifçilar, S.Alemdaroglu, I.Ayse Karaduman, A.Tunca Yilmaz, O.Bilgin, N.Sari, S.Chiriboga, C.Kane, S.Lee, J.Rome-Martin, D.Day, J.W.Beres, S.Duong, T.Gee, R.Dunaway Young, S.
Source
In: The Lancet Neurology. (The Lancet Neurology, January 2022, 21(1):42-52)
Subject
Language
English
ISSN
14744465
14744422