Généthon welcomes the first FDA-approved gene therapy for a neuromuscular disease

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). 

MYOCURE partner Généthon 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. The researchers demonstrated that a recombinant AAV9 vector made it possible to cross the blood-brain barrier and thus reach motor neurons, whose dysfunction is responsible for spinal muscular atrophy.

The CEO of Généthon, Frederic Revah, calls it a major breakthrough for patients and their families.

To read the full article, please visit genethon.fr