Welcome to AFM-Telethon's website
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).
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
The trial will be conducted in patients for whom pre-existing anti-AAV antibodies are currently a limitation to gene therapy treatment.
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!
