Find here all the latest news on AFM-Telethon and rare diseases.
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.
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.
€ 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.
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.
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.
The 10th International Limb-Girdle Muscular Dystrophy (LGMD) Awareness Day takes place on September 30. Organized by the LGMD Awareness Foundation, the day's mission is to draw attention to this group of rare neuromuscular diseases and their impact on the lives of affected children and adults in all regions of the world.
Alongside the hosts of France Televisions and surrounded by numerous artists, Mika will use all his talent, sensitivity and energy to support families in their fight against rare diseases. Discover his message !