Welcome to AFM-Telethon's website
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.
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.
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.
You live abroad and want to be part of the great Telethon adventure? Join us!
