Welcome to AFM-Telethon's website
At ASGCT 2026 in Boston, Genethon presented new clinical data from three of its gene therapy programs in Duchenne muscular dystrophy, gamma-sarcoglycanopathy (LGMD-R5), and Crigler-Najjar syndrome. In total, Genethon teams presented 8 oral presentations and 13 posters.
Atamyo Therapeutics presented the first safety, pharmacodynamics, and efficacy data from its ATA-200 gene therapy in LGMD-R5 at the ASGCT 2026 Annual Meeting. Results observed in the first treated patients showed high SGCG protein expression together with improvements in several biological and functional parameters
The discovery that the abnormal repetition of a short DNA sequence leads to the production of toxic proteins opens the way to new therapeutic approaches in oculopharyngodistal myopathy and oculopharyngeal myopathy with leukoencephalopathy.
New studies have been published on a lower-cost generic version of Spinraza in SMA, two therapies discontinued in Duchenne muscular dystrophy and home-based enzyme replacement therapy in Pompe disease.
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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