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The impossible becomes possible! Thanks to the momentum of the Telethon and the work of researchers, a true scientific and medical revolution has begun and the first victories against rare diseases - previously incurable - have been won.
We are pleased to announce that our 9th international congress dedicated to muscle biology and neuromuscular diseases, MYOLOGY 2027, will take place in STRASBOURG, France, on May 10-13, 2027. Don't miss the chance to be part of this major global gathering of myology experts!
Expert care pathways, new therapeutic approaches and clinical trial results… Here is an overview of recent studies shedding light on the management of myasthenia gravis and opening new perspectives for GNE myopathy and myositis.
On 17 and 18 June 2026, the GenoTher biocluster will bring together leading international experts in genetic medicine at the Maison de l’Océan in Paris for the GenoTher Summit 2026.
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.
Through its annual calls for proposals, each year AFM-Telethon supports new research projects in France and abroad, particularly for young researchers. After assessment by its Scientific advisory board, the Association finances the most relevant or most innovative initiatives in the development of therapeutic concepts and the understanding of the causes of rare and neuromuscular diseases. Discover its calls for proposals and financing opportunities.
Since 1987, the Telethon is a nearly thirty hours TV program broadcasted on the France Télévisions' channels. Discover all the ingrediants of the TV show.
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!
Sleep, treatments, natural history and symptom monitoring are highlighted in several neuromuscular diseases in this new research highlight.
Drugs in development or marketed with the support of AFM-Telethon concern neurological and neuromuscular diseases, diseases of the liver, vision, skin and immune system.
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.
For the 7 million people living with sickle cell disease worldwide, the authorisation granted by the French National Agency for Medicines and Health Products Safety (ANSM) to launch the DREPAMIR phase I/II clinical trial represents real hope. Supported by AFM-Téléthon and led by Professor Marina Cavazzana and Dr Anne Galy (ART-TG), the aim of this trial is to provide a long-term therapeutic solution for affected patients.