Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial

Mazurkiewicz-Bełdzińska M, Servais L, Baranello G, Boespflug-Tanguy O, Day JW, Deconinck NP, Klein A, Masson R, Mercuri EM, Rose KJ, Vlodavets DV, Xiong H, Zanoteli E, El-Khairi M, Gaki E, Kuthiala M, Gorni K, Kletzl H, Palfreeman L, Darras BT

The Lancet. Child & adolescent health