O. Schneider

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.

Normative data on spontaneous stride velocity, stride length, and walking activity in a non-controlled environment

D. Vissière, L. Servais, M. Demonceau, 2021, Orphanet Journal of Rare Diseases.

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

S. Corti, M. Pane, E. Mercuri, 2021, The Lancet Neurology.