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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
Baranello, Giovanni; Darras, Basil T.; Day, John W.; Deconinck, Nicolas; Klein, Andrea; Masson, Riccardo; Mercuri, Eugenio; Rose, Kristy; El-Khairi, Muna; Gerber, Marianne; Gorni, Ksenija; Khwaja, Omar; Kletzl, Heidemarie; Scalco, Renata S.; Seabrook, Timothy; Fontoura, Paulo; Servais, Laurent (2021). Risdiplam in Type 1 Spinal Muscular Atrophy. New England journal of medicine NEJM, 384(10), pp. 915-923. Massachusetts Medical Society MMS 10.1056/NEJMoa2009965
