Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial
Titel:
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial
Auteur:
Mercuri, Eugenio Deconinck, Nicolas Mazzone, Elena S Nascimento, Andres Oskoui, Maryam Saito, Kayoko Vuillerot, Carole Baranello, Giovanni Boespflug-Tanguy, Odile Goemans, Nathalie Kirschner, Janbernd Kostera-Pruszczyk, Anna Servais, Laurent Gerber, Marianne Gorni, Ksenija Khwaja, Omar Kletzl, Heidemarie Scalco, Renata S Staunton, Hannah Yeung, Wai Yin Martin, Carmen Fontoura, Paulo Day, John W