Welcome to AFM-Telethon's website
June 21 marks International Crigler-Najjar Syndrome Day. This rare genetic liver disease forces patients to live under intensive phototherapy. Thanks to a gene therapy developed by Généthon, the hope of a life without lamps is becoming a reality for some. But not all patients have access to it yet.
On June 20, for International Facioscapulohumeral Muscular Dystrophy (FSHD) Day, AFM-Téléthon reaffirms its commitment to individuals living with the disease and provides an update on recent research progress.
June 2 marks the European Myasthenia Gravis Day, an awareness initiative launched in 2023 by the European coalition ALL United for MG. This mobilisation brings together several patient organisations across Europe, including AFM-Téléthon. Its goals: to raise awareness of this rare disease, inform healthcare professionals, and actively support research efforts.
Presented on May 17 at the 2025 Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), the results of Genethon’s trial for Duchenne Muscular Dystrophy show stabilization of motor functions and a significant, sustained reduction in CPK levels in patients treated at the effective dose. The pivotal phase is scheduled to begin mid-2025 in Europe and the United States.
The French Muscular Dystrophy Association (AFM-Téléthon) is composed of patients and their families who are affected by genetic, rare and progressive diseases. Discover 60 years of fight.
You live abroad and want to be part of the great Telethon adventure? Join us!
