Release of the 73rd newsletter from the Institute

2024 Institute of Myology strategic project


Welcome to our 73rd newsletter.


A historic first: on May 24, the Food and Drug Administration approved Zolgensma®, a gene therapy drug for the treatment of spinal muscular atrophy. This is the first gene therapy authorized for a neuromuscular disease.


In France, an agreement was reached between the Biogen laboratory and the French state that allows access to Spinraza®, the first innovative therapeutic medicine offered to patients with spinal muscular atrophy type 1,2 and 3. The hospitals, in fact, will be entirely reimbursed for their costs.


And to that point, on May 28, the World Health Assembly of the World Health Organization was « seriously concerned » with prices of certain drugs and their consequences for patients. It adopted a resolution calling for more transparency on the actual prices of medicines and the determinants of their costs. The AFM-Telethon want concrete steps in this direction.


> Access the 73rd newletter from the Institute of Myology