Genethon welcomes the first FDA-approved gene therapy for a neuromuscular disease and emphasizes its decisive role in this historic step

On May 24, the Food and Drug Administration approved Zolgensma®, a gene therapy drug for the treatment of spinal muscular atrophy developed by AveXis (Novartis).
Genethon played a decisive role in the design of both the product and the route of administration of this first gene therapy for a neuromuscular disease, thanks to the pioneering work lead by Martine Barkats and her team.