It all began with the anger
Anger in the face of these rare diseases to be incurable, in the face of the helplessness of the medicine and the total absence of structure to which they can turn.
Created in 1958 by a dozen parents, the Association française contre les myopathies (AFM) has become an incredible war machine against rare diseases.
In 1987, she hosted the first Telethon French. The purpose of this? Collect 50 million francs to open a consultation multidisciplinary the hospital of the Pitié-Salpêtrière hospital (AP-HP) in Paris. But this year, the pledges will exceed, by far, the hopes of the wildest: 181 million francs were collected (27.6 million euros).
Thirty years later, the AFM-Telethon has participated in the establishment of all the links of the chain of production of drugs for gene therapy. The Figaro has traced image this epic (see the slideshow above). Four research laboratories have been created (the Généthon and Atlantic Gene Therapies for gene therapy, the Institute of myology for research on muscle, I-Stem for stem cells) as well as a platform from which medicines are developed (Yposkesi).
Since the first Telethon, the AFM has invested more than a billion euros in research and $ 700 million in aid to the families. The year 2017 has been marked by three scientific prowess: the realization of a first gene therapy trial in children with myopathy myotubulaire, the demonstration of the effectiveness of a gene to restore muscle strength in dogs with Duchenne muscular dystrophy, as well as the development of a patch of stem cells to treat retinitis pigmentosa, a rare disease of the vision.
The AFM-Telethon supports currently 33 trials in Humans in the course of, or in preparation for 26 different diseases of which 9 neuromuscular diseases and 17 other rare diseases (skin, blood, brain, vision, liver. ..
.. .. .).
17 are gene therapy trials, 7 cell therapy and 9 of pharmacology. The Telethon starts Friday and will run throughout the weekend.
To make your donations: 3637 or on www. .telethon.