Do a search on the AFM-Telethon website.
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.
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.
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.
Innovation in scientifical, medical, social and technological fields, AFM-Telethon is a pioneer in the research and support to improve patients’ lives. Three missions are at the heart of its action: Cure, Care, Communicate.
June 21 marks International Crigler-Najjar Syndrome Day. This rare genetic liver disease forces patients to live under intensive phototherapy. Thanks to a gene therapy developed by Généthon, the hope of a life without lamps is becoming a reality for some. But not all patients have access to it yet.
On June 20, for International Facioscapulohumeral Muscular Dystrophy (FSHD) Day, AFM-Téléthon reaffirms its commitment to individuals living with the disease and provides an update on recent research progress.