Find here all the latest news on AFM-Telethon and rare diseases.
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).
The trial will be conducted in patients for whom pre-existing anti-AAV antibodies are currently a limitation to gene therapy treatment.
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
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.
The 10th International Limb-Girdle Muscular Dystrophy (LGMD) Awareness Day takes place on September 30. Organized by the LGMD Awareness Foundation, the day's mission is to draw attention to this group of rare neuromuscular diseases and their impact on the lives of affected children and adults in all regions of the world.
Alongside the hosts of France Televisions and surrounded by numerous artists, Mika will use all his talent, sensitivity and energy to support families in their fight against rare diseases. Discover his message !
September 21, 2024 marks the 2nd World Myositis Day. At last year's World Myositis Congress in Berlin, international associations fighting inflammatory myopathies decided to dedicate the day to raising public awareness and mobilizing resources around these rare diseases. This worldwide initiative aims to better understand and treat myositis by uniting efforts on a global scale.
September 15, 2024 marks the 4th International Myotonic Dystrophies Awareness Day. This event, supported by over 60 organizations worldwide, aims to raise awareness among the general public, healthcare professionals and political decision-makers of the need to improve diagnosis, care and support for patients, and to speed up the development of medicines. The AFM-Telethon, which brings together French patients affected by these diseases, is committed to speeding up research. This new edition focuses on two specific areas in particular: raising awareness and educating clinical care teams, and preparing participants for clinical trials.
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 !
On Friday 31 August, 56 people from the Danish association MuskelsVindfonden, the Danish equivalent of AFM-Téléthon, visited the Institut de Myologie.
"Cure through Innovation" is a booklet that sums up the main actions and missions of the AFM-Téléthon association, as well as its key figures for 2023. The July 2024 edition has just been published!
On June 20, World FSHD Day reminds us of the importance of raising awareness and supporting research into facioscapulohumeral muscular dystrophy. Committed to the fight against FSHD, the AFM-Téléthon is making available a series of publications to enlight the advances in research by it, but also by many other neuromuscular diseases.
While it is customary for the heart of solidarity to beat for the Telethon on the first weekend in December, this year, exceptionally, the Telethon will be held on November 29 and 30, to make way for the ceremony to reopen the Cathedral of Notre-Dame de Paris.
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.
