Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment.
Mercuri E, Baranello G, Boespflug-Tanguy O, De Waele L, Goemans N, Kirschner J, Masson R, Mazzone ES, Pechmann A, Pera MC, Vuillerot C, Bader-Weder S, Gerber M, Gorni K, Hoffart J, Kletzl H, Martin C, McIver T, Scalco RS, Yeung WY, Servais L; SUNFISH Working Group.
Mercuri E, et al. Among authors: mciver t.
Eur J Neurol. 2023 Jul;30(7):1945-1956. doi: 10.1111/ene.15499. Epub 2022 Aug 1.
Eur J Neurol. 2023.
PMID: 35837793
Clinical Trial.