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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. 

A patient with Crigler–Najjar syndrome, immunised against the AAV vector used in gene therapy, was able to receive treatment as part of a Genethon-led trial, thanks to the prior administration of imlifidase, an enzyme developed by Hansa Biopharma.

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. 

To celebrate World Mitochondrial Disease Week, the “MitoGether” consortium, uniting 12 patient and family associations, is launching Mitogether.com. This new portal aims to become the leading online resource for everyone affected by a genetic mitochondrial disease: patients, families, healthcare professionals, researchers, and pharmaceutical industry stakeholders.

A new milestone has been reached in the clinical trial of gene therapy conducted by the Généthon laboratory for Duchenne muscular dystrophy.

Discover Cure Through Innovation 2025, 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 65 years of fighting.

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.  

Transparent and meticulous management: in 2024, AFM-Telethon committed €97.8 M to its social missions, in addition to €14 M in advances and investment for the Cure mission.  

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.

At each Telethon, thousands of volunteers and partners join forces to organize fundraising initiatives all over France and abroad. This participation is unique by its sheer scale.

Participation in the Telethon or support for our therapeutic and social innovation projects, your company can get involved in various ways. Find out how to become a partner/sponsor of AFM-Telethon.

AFM-Telethon is battling on all fronts, from access to diagnosis and appropriate treatment to close support, giving priority to the search for innovative solutions and a response to the needs of patients. 

Encouraging innovation, creating pathways between academic research and private research, supporting those involved in developing the drug: the AFM-Telethon is investing to speed up the arrival of innovative biotherapies. 

Treatments and therapeutic innovation are born out of the knowledge of genes and the understanding of the mechanisms causing disease. Beyond its three pioneering laboratories combined within the Biotherapies Institute for rare diseases, AFM-Telethon has been developing collaborations for many years with scientists from all over the world.

To accelerate the development of innovative treatments for rare diseases, the AFM-Telethon has created its own laboratories. Devoted to gene therapy of rare diseases (Genethon), to stem cells (I-Stem) and to myology (Institute of myology), they are now international leaders in their field. Located within the Institute for rare disease biotherapies, the Association’s three laboratories play a major role in the medical revolution.