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Documents to discover everything about the latest progress in research for a given disease or group of diseases. See the publications in English. 

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. 

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.

In this new research highlight, discover information on urinary disorders in myasthenia, results from the EMBARK trial in DMD, pregnancy in severe forms of SMA and the benefits of virtual reality during hospitalisation.

Committed to scientific research as well as to the support of patients and their families, AFM-Telethon acts independently, guided only by the urgency of the progressive disease, for the benefit of patients.  

Tested in three-dimensional muscle structures mimicking Duchenne muscular dystrophy, gene therapy improved muscle function without reducing the fibrosis characteristic of the disease.

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.