Welcome to AFM-Telethon's website
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.
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!
Patient care and research in mitochondrial myopathies caused by thymidine kinase 2 (TK2) deficiency are currently advancing on several fronts, with the possible arrival of a first therapy in Europe for early-onset forms and improved monitoring of disease progression in late-onset forms.
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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