Généthon on the verge of finding a treatment for Duchenne muscular dystrophy

Seeing little Sacha, Telethon ambassador, running down the stairs, it’s hard to imagine that he couldn’t run or jump just two years ago. At 8 years old, he is one of five children suffering from Duchenne muscular dystrophy to have benefited, in 2022, from an experimental treatment developed by Généthon, the research center of the AFM-Téléthon, which focuses on developing gene therapy drugs for rare diseases. Duchenne muscular dystrophy affects 1 in 5,000 births in France, almost all of them boys.

It causes progressive degeneration of muscles. At the age of 12, children are forced to use wheelchairs. Most die between the ages of 20 and 40. But the AFM-Téléthon hopes to have finally found a treatment that is not limited to alleviating the symptoms of the disease. The association has been working on the subject for twenty years, starting with its creation in 1958. « Nine parents…