79,801,520 euros : the battle goes on !
Mika, patron of the 2024 Telethon, made a heartfelt appeal for public generosity at the end of the show. Speaking to viewers from 200 countries, he encouraged everyone to make a donation on telethon.fr
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Genethon and Hansa Biopharma launch a phase 2 clinical trial for Crigler-Najjar syndrome
The trial will be conducted in patients for whom pre-existing anti-AAV antibodies are currently a limitation to gene therapy treatment.
Genethon Presents Promising Results for Gene Therapy in DMD
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 plans to launch a pivotal trial in Europe and the United States in 2025.
World Duchenne Awareness Day: Genethon at the forefront of the fight against Duchenne muscular dystrophy
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 first patients to be treated. Discover our video of patient testimonials !
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Our fight to cure
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Change my preferences60 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.
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