FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 spinal muscular atrophy (SMA) (P7-9.009)

Darras B, Baranello G, Boespflug-Tanguy O, Day J, Deconinck N, Klein A, Masson R, Mazurkiewicz-Beldzinska M, Mercuri E, Rose K, Vlodavets D, Xiong H, Zanoteli E, El-Khairi M, Gerber M, Gorni K, Kletzl H, Palfreeman L, Dodman A, Servais L

Neurology