Virginie Germa

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

G. Comi, S. Corti, E. Mazzone, 2022, The Lancet Neurology.

Palliative Care in SMA Type 1: A Prospective Multicenter French Study Based on Parents' Reports

F. Rivier, P. Sabouraud, C. Barnérias, 2020, Frontiers in Pediatrics.