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Discover updates on Duchenne muscular dystrophy, spinal muscular atrophy and progressive motor disorders such as Kennedy disease.

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.

Lines of research moving forward, lines of solidarity expanding, lines of resistance giving way to treatments. May this new year be driven by scientific and medical advances, stronger solidarity and new victories against disease. AFM-Téléthon wishes a happy 2026 to all those who refuse the impossible and are building a true revolution in medicine!

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.

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.

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.

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.

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

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.