News

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. Read more

€ 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 … [Read more]

For the 7 million people living with sickle cell disease worldwide, the authorisation granted by the French National Agency for Medicines and Health Products Safety (ANSM) to launch the DREPAMIR phase I/II clinical trial represents real hope. Supported by AFM-Téléthon and led by Professor Marina Cavazzana and Dr Anne Galy (ART-TG), the aim of this … [Read more]

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 … [Read more]

AFM-Téléthon and The Muscular Dystrophy Association (MDA)  are proud to announce the awarding of two cutting-edge research grants to accelerate the development of treatments for mitochondrial myopathies and amyotrophic lateral sclerosis (ALS). “This partnership with MDA is an essential step to build a strong collaboration between our two associations, through the support of these two … [Read more]

The trial will be conducted in patients for whom pre-existing anti-AAV antibodies are currently a limitation to gene therapy treatment. Genethon, the Telethon laboratory, and Hansa Biopharma, a pioneer in enzyme technology for rare immunological diseases, today announced the launch of a phase 2 trial in patients suffering from Crigler-Najjar disease, a rare liver disorder, … [Read more]

Genethon, the pioneering gene therapy research laboratory created by AFM-Telethon, today presented positive results from the Phase 1/2 dose escalation part of the trial evaluating its gene therapy, GNT0004, for Duchenne muscular dystrophy (DMD) at the ASGCT Breakthroughs in Muscular Dystrophy conference, Nov. 19 – 20, 2024, in Chicago, IL. Based on these results, Genethon … [Read more]

World Duchenne Muscular Dystrophy Day provides an opportunity to note the acceleration in the development of innovative therapies for this neuromuscular disease, which is the most common in children. Gene therapy is one of the most promising avenues. Genethon, the AFM-Telethon laboratory, is currently developing a drug candidate which has demonstrated its efficacy in the … [Read more]

The initial results of Genethon’s gene therapy trial for Duchenne muscular dystrophy were presented on Tuesday 23 April at the Myology 2024 congress in Paris. Discover the results

92,905,533 is the final amount raised by the Telethon held on 8 and 9 December 2023. This exceptional result bears witness to the unfailing mobilization of millions of donors, volunteers, and partners throughout France and abroad. It is also the result of the commitment of our patron, Vianney, as well as the artists, presenters, and … [Read more]