Find here all the latest news on AFM-Telethon and rare diseases.
More than ever, the French people said “Yessss” to the Téléthon! The exceptional Téléthon 2025 fundraising total reflects the remarkable trust and loyalty of volunteers, donors and partners committed alongside us. A huge thank you to each and every one of you!
An internationally recognized expert in the field of immunology and the development of gene therapies, Klaudia Kuranda is joining the AFM-Téléthon laboratories to establish and lead a new Immunology Center that will bring together the complementary expertise of Genethon, a leading gene therapy non-profit biotech organization, and the Institute of Myology, a center of expertise for muscle and muscle disorders.
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.
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.
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.
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.