Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain.
Cornell N, Childs AM, Wraige E, Munot P, Ambegaonkar G, Chow G, Hughes I, Illingworth M, Majumdar A, Marini-Bettolo C, Parasuraman D, Spinty S, Willis T, Scoto M, Baranello G; Paediatric UK Risdiplam EAMS Working Group.
Cornell N, et al. Among authors: chow g.
J Neuromuscul Dis. 2024;11(2):361-368. doi: 10.3233/JND-230162.
J Neuromuscul Dis. 2024.
PMID: 38189761
Free PMC article.