Welcome to AFM-Telethon's website
More than ever, the French people said “Yessss” to the Téléthon! The exceptional Téléthon 2025 fundraising total reflects the remarkable trust and loyalty of volunteers, donors and partners committed alongside us. A huge thank you to each and every one of you!
An internationally recognized expert in the field of immunology and the development of gene therapies, Klaudia Kuranda is joining the AFM-Téléthon laboratories to establish and lead a new Immunology Center that will bring together the complementary expertise of Genethon, a leading gene therapy non-profit biotech organization, and the Institute of Myology, a center of expertise for muscle and muscle disorders.
In spinal muscular atrophy linked to SMN1 (SMA), a single dose of a novel gene therapy called GC101 improved motor function in nine patients with type II and type III SMA aged six months to 18 years.
In this Research Highlights issue, we present recent study findings on thymectomy and CAR-T cell therapy in myasthenia, as well as the evaluation of ChatGPT for supporting rehabilitation in adolescents with myositis.
The French Muscular Dystrophy Association (AFM-Téléthon) is a patient-led organisation driving research and support for people with rare genetic diseases. From groundbreaking therapies to daily care, discover how AFM-Téléthon turns innovation into hope.
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