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Crigler-Najjar
Actualité
21/06/2025

International Crigler-Najjar Day: A Treatment in Trial

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.

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Le 20 juin, à l’occasion de la Journée internationale de la dystrophie facio-scapulo-humérale (FSHD), l’AFM-Téléthon réaffirme son engagement aux côtés des personnes concernées et fait le point sur les progrès de la recherche.
Actualité
20/06/2025

International FSHD Day: Where Does Research Stand?

On June 20, for International Facioscapulohumeral Muscular Dystrophy (FSHD) Day, AFM-Téléthon reaffirms its commitment to individuals living with the disease and provides an update on recent research progress.

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AFM-Telethon-progress-in-research-booklets
Page
19/06/2025

Advances in research 

Documents to discover everything about the latest progress in research for a given disease or group of diseases. See the publications in English. 

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June 2 marks the European Myasthenia Gravis Day, an awareness initiative launched in 2023 by the European coalition ALL United for MG. This mobilisation brings together several patient organisations across Europe, including AFM-Téléthon.
Actualité
02/06/2025

June 2: Myasthenia in the Spotlight – Disease, Research, Commitment

June 2 marks the European Myasthenia Gravis Day, an awareness initiative launched in 2023 by the European coalition ALL United for MG. This mobilisation brings together several patient organisations across Europe, including AFM-Téléthon. Its goals: to raise awareness of this rare disease, inform healthcare professionals, and actively support research efforts.

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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.
Actualité
21/05/2025

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.

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Thérapie génique : Résultats consolidés jusqu’à deux ans de l’essai de Généthon pour la myopathie de Duchenne
Actualité
19/05/2025

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.

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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
Actualité
16/05/2025

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.

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AFM-Telethon supports research throug annual calls for proposals
Appel d'offre
02/04/2025

Calls for proposals

Through its annual calls for proposals, each year AFM-Telethon supports new research projects in France and abroad, particularly for young researchers. After assessment by its Scientific advisory board, the Association finances the most relevant or most innovative initiatives in the development of therapeutic concepts and the understanding of the causes of rare and neuromuscular diseases. Discover its calls for proposals and financing opportunities. 

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Le parrain Mika et les familles ambassadrices sur le plateau du Téléthon 2024
Actualité
31/03/2025

€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.

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Vignette Actualité - Visite d'un groupe dans les laboratoires d'ISTEM le 13 mars 2025
Actualité
24/03/2025

LGMD R2 : new pharmacological approaches

A joint study conducted by I-Stem, Genethon and the Institute of Myology reveals the positive effects of two drugs—saracatinib and bazedoxifene—on muscle cell models of dysferlin-related limb-girdle muscular dystrophy type R2. This opens up a novel research avenue.

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Grâce au soutien financier de l’AFM-Téléthon, une étape décisive vient d’être franchie dans la lutte contre la drépanocytose.
Actualité
05/03/2025

Sickle cell anemia: AFM-Telethon supports gene therapy trial

Thanks to the financial support of the AFM-Téléthon, a decisive step has just been taken in the fight against sickle cell disease. In collaboration with AP-HP, Institut Imagine and SK pharmteco, the AFM- Telethon will enable the production of lentiviral vectors for the development of a gene therapy clinical trial. This represents hope for the 5 million patients worldwide affected by this incurable blood disease. 

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Lucie sauvée par une thérapie génique issue des recherches menées à Généthon
Page
25/02/2025

AFM-Telethon life-changing treatments

The impossible becomes possible! Thanks to the momentum of the Telethon and the work of researchers, a true scientific and medical revolution has begun and the first victories against rare diseases - previously incurable - have been won.  

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