Un garçon en visite dans le laboratoire Généthon en compagnie d'une chercheuse

AFM-Telethon

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Thérapie génique : Résultats consolidés jusqu’à deux ans de l’essai de Généthon pour la myopathie de Duchenne

Gene Therapy: Two-Year Consolidated Results from Genethon’s Clinical Trial for Duchenne Muscular Dystrophy

Presented on May 17 at the 2025 Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), the results of Genethon’s trial for Duchenne Muscular Dystrophy show stabilization of motor functions and a significant, sustained reduction in CPK levels in patients treated at the effective dose. The pivotal phase is scheduled to begin mid-2025 in Europe and the United States.

Le 11 juin 2025, Généthon, le laboratoire de l’AFM-Téléthon, co-organise à Évry le GenoTher Summit 2025, un événement scientifique d’envergure internationale qui rassemblera l’écosystème de la thérapie génique autour d’un objectif commun : accélérer le développement et l’industrialisation de traitements innovants pour les maladies rares et au-delà.

GenoTher Summit 2025: An International Summit to Sharpen the Future of Gene Therapy

On June 11, 2025, Généthon, the AFM-Téléthon laboratory, is co-organizing the GenoTher Summit 2025 in Évry, a major international scientific event bringing together the gene therapy ecosystem with a shared ambition: sharpening the strategic trajectory and industrial readiness of innovative treatments for rare diseases and beyond.

Les résultats de l’essai européen MIROCALS, soutenu par l'AFM-Téléthon et Généthon, apportent un nouvel éclairage sur une piste thérapeutique prometteuse pour la Sclérose Latérale Amyotrophique (SLA

ALS: The MIROCALS Trial Paves the Way for a New Treatment

The results of the European MIROCALS trial provide new insights into a promising therapeutic approach for Amyotrophic Lateral Sclerosis (ALS). Low-dose interleukin-2 could slow disease progression in certain patients. A study supported by AFM-Téléthon and its laboratory, Généthon, through its biobank.

Le parrain Mika et les familles ambassadrices sur le plateau du Téléthon 2024

€96,553,593 – an outstanding result for Telethon 2024!

€ 96, 553, 593 : this is the final amount raised during the Telethon held on 29 and 30 November 2024. This result reflects an extraordinary outpouring of solidarity, driven by millions of donors, volunteers and partners who have stood by families in their fight for nearly 40 years. It is also the outcome of the outstanding commitment of our patron, Mika, and the many presenters and professionals from France Télévisions, who powerfully amplified the voices of families and researchers united in their determination to defeat the disease.

Our fight to cure

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60 years of fight against diseases

The French Muscular Dystrophy Association (AFM-Téléthon) is composed of patients and their families who are affected by genetic, rare and progressive diseases. Discover 60 years of fight.

Join the Telethon for french people abroad

You live abroad and want to be part of the great Telethon adventure? Join us!