Do a search on the AFM-Telethon website.
In spinal muscular atrophy linked to SMN1 (SMA), a single dose of a novel gene therapy called GC101 improved motor function in nine patients with type II and type III SMA aged six months to 18 years.
In this Research Highlights issue, we present recent study findings on thymectomy and CAR-T cell therapy in myasthenia, as well as the evaluation of ChatGPT for supporting rehabilitation in adolescents with myositis.
Antwerp put the spotlight on CMT by hosting the second European congress dedicated to Charcot-Marie-Tooth disease. The event strengthened cooperation across Europe among researchers, clinicians and patients. Discover the key highlights: promising treatments and diagnostic innovations.
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.
The thirty hours of Téléthon 2025 come to an end with a total of 83,504,259 euros! A result worthy of Santa’s achievements, an exceptional patron thanks to her energy and her determination to “break the records”. This result embodies the trust and loyalty of donors standing alongside families, researchers and volunteers. Today, a new medicine driven by the Téléthon is changing and saving lives. This fight must continue, as 95% of rare diseases remain without treatments.
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.
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.