After 25 years of research supported by the Telethon, the first treatments for rare and neuromuscular diseases are seeing the light of day. The French Muscular Dystrophy Association (AFM-Téléthon) has adopted a strategy which benefits as many people as possible: to use models of rare diseases to explore therapeutic approaches which are sometimes also applicable to common diseases.
Innovations for patients
Through Telethon donations, AFM-Téléthon does not only explore innovative approaches to treating rare diseases. It also fights so that patients can be a full part of society. Telethon donations also help develop innovative products which enable handicapped people to carry out their daily activities again.
Biotherapies Institute for Rare Diseases
Founded in 2011, the Institute for biotherapies for rare diseases gathers the Institute of Myology, Généthon and I-Stem. An initiative absolutely unique in the world.
Call for proposals
AFM-Téléthon supports projects following Call for Proposals after review by its Scientific Council or by Committees of Experts. Composed of volunteer scientists and clinicians, AFM-Téléthon´s Scientific Council is an advisory organ and also makes proposals.
In order to develop innovative treatments you must have the right tools: vector production centres, screening platforms, databases and tissue banks.
Developing innovative therapies
Correcting a defective gene, inserting a gene-drug, grafting cells capable of regenerating healthy tissue: AFM-Téléthon paves the way for the medicine of the future and supports nearly 40 human trials.