The impossible becomes possible! Thanks to the momentum of the Telethon and the work of researchers, a true scientific and medical revolution has begun and the first victories against rare diseases - previously incurable - have been won.
From genome maps to the discovery of the genes responsible for diseases, an entire sector of world medicine has made a spectacular leap forward, thanks to the determination of AFM-Telethon families, and the momentum of the Telethon.
Gene, cellular and pharmacogenetic therapy: AFM-Telethon uses donations to develop new therapies for the most frequent rare diseases.
Peeling an apple. Lifting your arms. Walking. Smiling. Breathing. These routine actions use muscles and we accomplish them without thinking. But with neuromuscular diseases, the muscles are attacked and movement is prevented. In order to better treat them, AFM-Telethon wants to make myology a separate discipline.
While the first gene therapy drugs are obtaining marketing authorization and being made available to patients, the challenge of industrializing their production is a crucial one.