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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; ... (2022). Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial. The lancet. Neurology, 21(12), pp. 1110-1119. Elsevier 10.1016/S1474-4422(22)00339-8
Darras, Basil T; Masson, Riccardo; Mazurkiewicz-Bełdzińska, Maria; Rose, Kristy; Xiong, Hui; Zanoteli, Edmar; Baranello, Giovanni; Bruno, Claudio; Vlodavets, Dmitry; Wang, Yi; El-Khairi, Muna; Gerber, Marianne; Gorni, Ksenija; Khwaja, Omar; Kletzl, Heidemarie; Scalco, Renata S; Fontoura, Paulo; Servais, Laurent (2021). Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls. The New England journal of medicine, 385(5), pp. 427-435. Massachusetts Medical Society 10.1056/NEJMoa2102047
