It is a rare disease emblematic of Telethon whose 38th edition takes place from this Friday : Duchenne muscular dystrophy. In 1958, she was behind the creation of the AFM, the association which organizes the charity event to finance research into genetic diseases. Until now deemed incurable, researchers have developed a promising treatment to combat this pathology which destroys muscles and affects one boy in 3,000 in France.
Five children aged 6 to 10 benefited from a pioneering drug that allowed them to rebuild muscle, without significant side effects. “The word impossible no longer exists with this advance, this extraordinary breakthrough”, rejoices Laurence Tiennot-Herment, president of the French Association against myopathies (AFM), guest of Ma France on France Bleu this Friday.
“We say to ourselves, that’s it, it works!”
Young Sacha, 8 years old, was able to benefit from this treatment during a clinical trial. “When we see Sacha climbing these stairs one step after the other, these are moments of intense joy,” says the president of the AFM, while “in Duchenne muscular dystrophy, we tend to see children clinging to the bars.” “We say to ourselves, that’s it, it works!” she confides while recognizing that “It works on some patients.” From now on, “the challenge of the Telethon is to validate [le traitement] on many more children. This objective is considered to be “a race against time” par Laurence Tiennot-Herment, “but we are winning it”.
Presented at two medical congresses, the treatment will be administered in the first quarter of 2025 to around sixty children in France, the United States and the United Kingdom, with the aim of commercialization in “2027-2028”, according to the AFM. “A victory over one disease is a victory for many others”assures Laurence Tiennot-Herment. If the effectiveness of treatment for Duchenne muscular dystrophy is confirmed, “It’s a huge breakthrough for a number of diseases that have the same mechanisms as Duchenne muscular dystrophy. It’s a snowball effect.” The president of the AFM also recalls that 95% of rare diseases do not yet have treatment.
Mathieu Génon / OKO
The Telethon “benefits everyone”
On France Bleu, Laurence Tiennot-Herment recalls that the Telethon can benefit “to everyone”. These advances in rare diseases “open the way for common diseases”she explains. In particular, she takes the example of “all the innovations on rare retinal diseases” Who “allow there to be trials of innovative therapy for age-related macular degeneration which affects 20% of the population”. The president of the AFM affirms that the Telethon has continued to evolve: “Initially, it was the fight against Duchenne muscular dystrophy, then the fight against muscular dystrophy, the fight against 7,000 rare diseases and today it concerns everyone.
