Do a search on the AFM-Telethon website.
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.
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 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.
Through its annual calls for proposals, each year AFM-Telethon supports new research projects in France and abroad, particularly for young researchers. After assessment by its Scientific advisory board, the Association finances the most relevant or most innovative initiatives in the development of therapeutic concepts and the understanding of the causes of rare and neuromuscular diseases. Discover its calls for proposals and financing opportunities.
€ 96, 553, 593 : this is the final amount raised during the Telethon held on 29 and 30 November 2024. This result reflects an extraordinary outpouring of solidarity, driven by millions of donors, volunteers and partners who have stood by families in their fight for nearly 40 years. It is also the outcome of the outstanding commitment of our patron, Mika, and the many presenters and professionals from France Télévisions, who powerfully amplified the voices of families and researchers united in their determination to defeat the disease.
Documents to discover everything about the latest progress in research for a given disease or group of diseases. See the publications in English.
A joint study conducted by I-Stem, Genethon and the Institute of Myology reveals the positive effects of two drugs—saracatinib and bazedoxifene—on muscle cell models of dysferlin-related limb-girdle muscular dystrophy type R2. This opens up a novel research avenue.
Thanks to the financial support of the AFM-Téléthon, a decisive step has just been taken in the fight against sickle cell disease. In collaboration with AP-HP, Institut Imagine and SK pharmteco, the AFM- Telethon will enable the production of lentiviral vectors for the development of a gene therapy clinical trial. This represents hope for the 5 million patients worldwide affected by this incurable blood disease.
The impossible becomes possible! Thanks to the momentum of the Telethon and the work of researchers, a true scientific and medical revolution has begun and the first victories against rare diseases - previously incurable - have been won.
Drugs in development or marketed with the support of AFM-Telethon concern neurological and neuromuscular diseases, diseases of the liver, vision, skin and immune system.
AFM-Téléthon and The Muscular Dystrophy Association (MDA) are proud to announce the awarding of two cutting-edge research grants to accelerate the development of treatments for mitochondrial myopathies and amyotrophic lateral sclerosis (ALS).
The trial will be conducted in patients for whom pre-existing anti-AAV antibodies are currently a limitation to gene therapy treatment.
Mika, patron of the 2024 Telethon, made a heartfelt appeal for public generosity at the end of the show. Speaking to viewers from 200 countries, he encouraged everyone to make a donation on telethon.fr
Genethon, the pioneering gene therapy research laboratory created by AFM-Telethon, today presented positive results from the Phase 1/2 dose escalation part of the trial evaluating its gene therapy, GNT0004, for Duchenne muscular dystrophy (DMD) at the ASGCT Breakthroughs in Muscular Dystrophy conference, Nov. 19 - 20, 2024, in Chicago, IL. Based on these results, Genethon plans to launch a pivotal trial in Europe and the United States in 2025.
A look at clinical trials underway or in preparation in France and worldwide.