Welcome to AFM-Telethon's website
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.
On June 11, 2025, Généthon, the AFM-Téléthon laboratory, is co-organizing the GenoTher Summit 2025 in Évry, a major international scientific event bringing together the gene therapy ecosystem with a shared ambition: sharpening the strategic trajectory and industrial readiness of innovative treatments for rare diseases and beyond.
The results of the European MIROCALS trial provide new insights into a promising therapeutic approach for Amyotrophic Lateral Sclerosis (ALS). Low-dose interleukin-2 could slow disease progression in certain patients. A study supported by AFM-Téléthon and its laboratory, Généthon, through its biobank.
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.
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