Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo‐controlled, dose‐finding trial followed by 24 months of treatment

Mercuri, Eugenio ; Baranello, Giovanni ; Boespflug‐Tanguy, Odile ; De Waele, Liesbeth ; Goemans, Nathalie ; Kirschner, Janbernd ; Masson, Riccardo ; Mazzone, Elena S. ; Pechmann, Astrid ; Pera, Maria Carmela ; Vuillerot, Carole ; Bader‐Weder, Silvia ; Gerber, Marianne ; Gorni, Ksenija ; Hoffart, Janine ; Kletzl, Heidemarie ; Martin, Carmen ; McIver, Tammy ; Scalco, Renata S. ; Yeung, Wai Yin ; Servais, Laurent

European journal of neurology, 2023-07, Vol.30 (7), p.1945-1956 [Periódico revisado por pares]

England: John Wiley and Sons Inc

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