Search

Genethon, the pioneering gene therapy research laboratory created by AFM-Telethon, today presented positive results from the Phase 1/2 dose escalation part of the trial evaluating its gene therapy, GNT0004, for Duchenne muscular dystrophy (DMD) at the ASGCT Breakthroughs in Muscular Dystrophy conference, Nov. 19 - 20, 2024, in Chicago, IL. Based on these results, Genethon plans to launch a pivotal trial in Europe and the United States in 2025. 

A look at clinical trials underway or in preparation in France and worldwide.

Created in 1987 at the initiative of Bernard Barataud and Pierre Birambeau, both fathers of sons suffering grom Duchenne muscular dystrophy, the Telethon combines, since its first edition, a TV program and dozen of thousands of events throughout France. Discover the birth of this unique fundraising event.  

AFM-Telethon wanted to formalise the objectives that clinical study protocols should pursue and the points of attention they call for concerning organisational and methodological aspects. This formalisation should help AFM members in their participation in protocols.

Phone number: + 33 1 69 47 28 28

Postal address:
Association Française contre les Myopathies,
1, rue de l’Internationale,
91002 Evry,
France

You are now on a website that is edited by the French Muscular Dystrophy Association (AFM-Téléthon), a 1901 law association of public utility. Its headquarters are based in Paris : AFM-Institut de Myologie, 47-83 boulevard de l’Hôpital, 75651 Paris Cedex 13.

From formerly unknown diseases to conquered diseases, a medical revolution is underway and victories are multiplying.

Find here information on diseases : advances of research booklets presenting the lastest information on a given disease or group of diseases, information on trials and access to the Myobase portal. 

AFM-Telethon has long struggled to defend the rights of patients, of people living with disabilities, and to obtain recognition for rare diseases. As a driver of research and innovation, it defends an ambitious national policy.  

The AFM-Telethon has chosen to implement a strategy of public interest to benefit as many people as possible.  

While the first gene therapy drugs are obtaining marketing authorization and being made available to patients, the challenge of industrializing their production is a crucial one.

Peeling an apple. Lifting your arms. Walking. Smiling. Breathing. These routine actions use muscles and we accomplish them without thinking. But with neuromuscular diseases, the muscles are attacked and movement is prevented. In order to better treat them, AFM-Telethon wants to make myology a separate discipline. 

The French Telethon also takes place abroad. French expatriates can join in this 30-hour marathon of celebration and solidarity from all over the world. You live abroad and want to be part of the great Telethon adventure? Join us!  

You can pass on not only your genes It is above all about deep convictions, to last beyond your lifetime.  

Gene, cellular and pharmacogenetic therapy: AFM-Telethon uses donations to develop new therapies for the most frequent rare diseases.