학술논문
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial
Document Type
Article
Author
Volpe, Joseph J; Posner, John; Kellner, Ulrich; Quinlivan, Rosaline; Gerber, Marianne; Khwaja, Omar; Scalco, Renata S; Seabrook, Timothy; Koch, Armin; Balikova, Irina; Joniau, Inge; Accou, Geraldine; Tahon, Valentine; Wittevrongel, Sylvia; De Vos, Elke; de Holanda Mendonça, Rodrigo; Matsui Jr, Ciro; Fornazieri Darcie, Ana Letícia; Machado, Cleide; Kiyoko Oyamada, Maria; Martini, Joyce; Polido, Graziela; Rodrigues Iannicelli, Juliana; Caires de Oliveira Achili Ferreira, Juliana; Hu, Chaoping; Zhu, Xiaomei; Qian, Chen; Shen, Li; Li, Hui; Shi, Yiyun; Zhou, Shuizhen; Xiao, Ying; Zhou, Zhenxuan; Wang, Sujuan; Sang, Tian; Wei, Cuijie; Dong, Hui; Cao, Yiwen; Wen, Jing; Li, Wenzhu; Qin, Lun; Barisic, Nina; Celovec, Ivan; Galiot Delic, Martina; Ivkic, Petra Kristina; Vukojevic, Nenad; Kern, Ivana; Najdanovic, Boris; Skugor, Marin; Tomas, Josipa; Boespflug-Tanguy, Odile; De Lucia, Silvana; Seferian, Andrea; Barreau, Emmanuel; Mnafek, Nabila; Peche, Helene; Grange, Allison; Trang Nguyen, Diem; Milascevic, Darko; Tachibana, Shotaro; Pagliano, Emanuela; Bianchi Marzoli, Stefania; Santarsiero, Diletta; Garcia Sierra, Myriam; Tremolada, Gemma; Arnoldi, Maria Teresa; Vigano, Marta; Dosi, Claudia; Zanin, Riccardo; Schembri, Veronica; Brolatti, Noemi; Rao, Giuseppe; Tassara, Elisa; Morando, Simone; Tacchetti, Paola; Pedemonte, Marina; Priolo, Enrico; Sposetti, Lorenza; Comi, Giacomo Pietro; Govoni, Alessandra; Osnaghi, Silvia Gabriella; Minorini, Valeria; Abbati, Francesca; Fassini, Federica; Foa, Michaela; Lopopolo, Amalia; Pane, Marika; Palermo, Concetta; Pera, Maria Carmela; Amorelli, Giulia Maria; Barresi, Costanza; D'Amico, Guglielmo; Orazi, Lorenzo; Coratti, Giorgia; Leone, Daniela; Laura, Antonaci; De Sanctis, Roberto; Berti, Beatrice; Kimura, Naoki; Takeshima, Yasuhiro; Shimomura, Hideki; Lee, Tomoko; Gomi, Fumi; Morimatsu, Takanobu; Furukawa, Toru; Stodolska-Koberda, Urszula; Waskowska, Agnieszka; Kolendo, Jagoda; Sobierajska-Rek, Agnieszka; Modrzejewska, Sandra; Lemska, Anna; Melnik, Evgenia; Artemyeva, Svetlana; Leppenen, Natalya; Yupatova, Nataliya; Monakhova, Anastasya; Papina, Yulia; Shidlovsckaia, Olga; Litvinova, Elena; Enzmann, Cornelia; Galiart, Elea; Gugleta, Konstantin; Wondrusch Haschke, Christine; Topaloglu, Haluk; Oncel, Ibrahim; Ertugrul, Nesibe Eroglu; Konuskan, Bahadir; Eldem, Bora; Kadayifçilar, Sibel; Alemdaroglu, Ipek; Sari, Seher; Bilgin, Neslihan; Karaduman, Aynur Ayse; Sarikaya, Fatma Gokcem Yildiz; Graham, Robert J; Ghosh, Partha; Casavant, David; Levine, Alexis; Titus, Rachael; Engelbrekt, Amanda; Ambrosio, Lucia; Fulton, Anne; Baglieri, Anna Maria; Dias, Courtney; Maczek, Elizabeth; Pasternak, Amy; Beres, Shannon; Duong, Tina; Gee, Richard; Young, Sally; Masson, Riccardo ; Mazurkiewicz-Bełdzińska, Maria; Rose, Kristy; Servais, Laurent; Xiong, Hui; Zanoteli, Edmar; Baranello, Giovanni; Bruno, Claudio; Day, John W; Deconinck, Nicolas; Klein, Andrea; Mercuri, Eugenio; Vlodavets, Dmitry; Wang, Yi; Dodman, Angela; El-Khairi, Muna; Gorni, Ksenija; Jaber, Birgit; Kletzl, Heidemarie; Gaki, Eleni; Fontoura, Paulo; Darras, Basil T
Source
In The Lancet Neurology December 2022 21(12):1110-1119
Subject
Language
ISSN
1474-4422