Terapia génica
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial
The Lancet Neurology Fecha de publicación: Autores: Prof Eugenio Mercuri, MD , Prof Francesco Muntoni, MD, Giovanni Baranello, MD, Riccardo Masson,...
