Research into rare diseases funded by AFM-Téléthon
Although each of the 6,000 to 7,000 rare diseases has its own specific problems, they all share the common fact that there is no curative treatment, diagnosis is difficult to obtain as is access to care and, in the end, these patients are excluded from social life. A cross-cutting and global vision is essential if effective research and drug development policies are to be implemented.
Ever since the first Telethon was launched, spectacular progress has been made thanks to the funds that were collected, including discoveries for many rare diseases. Over 80% of them have a genetic origin.
Trials and skills centers
Apart from neuromuscular diseases, AFM-Téléthon is heavily involved in research into rare diseases, real models for the development of innovative treatments. It is also an unusual and unique association, that produces and tests its own gene therapy drugs on a semi-industrial scale, through Généthon Bioprod.