Latest news

Lamotrigine could represent an alternative to mexiletine for the treatment of non-dystrophic myotonias, including over the long term.

While gene therapy is currently administered intravenously in infants with spinal muscular atrophy (SMA), two recent clinical trials show that it is also effective when delivered intrathecally in older patients.

In this new research highlight, discover information on executive functions in Duchenne muscular dystrophy, the development of new gene therapy vectors and a study on lactate measurement.

Alpelisib, RK783 and KTI-2338 have shown efficacy in laboratory studies in fibrodysplasia ossificans progressiva.

Discover in this new research highlight information on predictive factors for the progression of a form of myositis, the role of complement in myasthenia, and recent work on a form of mitochondrial myopathy.

Treatment with apitegromab, which acts directly on muscles, appears to improve motor function in patients already treated with Spinraza or Evrysdi, according to a trial involving nearly 200 patients with type II or III spinal muscular atrophy.

The European Commission has just approved an extension for higher doses of nusinersen (Spinraza) in spinal muscular atrophy.

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.

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.