Find here all the latest news on AFM-Telethon and rare diseases.
A trial conducted at Papa Giovanni XXIII Hospital in Bergamo, within the international CureCN consortium and sponsored by Généthon, demonstrates the long-term benefits of gene therapy for an extremely rare and potentially life-threatening liver disease that forces patients to undergo phototherapy every night and may require a liver transplant.
AFM-Telethon is launching a two-year strategic research programme with Professor Drew Weissman, recipient of the 2023 Nobel Prize in Physiology or Medicine for his groundbreaking work on messenger RNA, and Professor James Dowling, a leading expert in neuromuscular diseases, at the University of Pennsylvania. The aim is to develop lipid nanoparticles capable of targeting muscle tissue while minimising uptake by the liver.
Research into glycogen storage disease type III is gaining momentum, with advances across many areas and active contributions from Généthon and I-Stem.
A new design, enriched content, simpler, smoother and more intuitive navigation, and an innovative AI-powered search engine… The Institute of Myology has completely redesigned its website to provide users with a more seamless and effective browsing experience. The new site makes the expertise and knowledge of this centre dedicated to muscle and muscle diseases more accessible than ever.
The 9th international congress dedicated to muscle biology and neuromuscular diseases, MYOLOGY 2027, will take place in Strasbourg, France, from May 10 to 13, 2027. Registration and abstract submissions are now open on the dedicated website.
Discover Cure Through Innovation 2026, the reference brochure presenting at a glance the core missions, commitments, and achievements of AFM-Téléthon. An essential document to grasp the diversity of its actions and the emergence of an innovative medicine born from 66 years of fighting.
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.
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.