The Telethon launches its 38th edition on Friday, against a backdrop of strong hope for Duchenne muscular dystrophy and increased financial needs

Finally a solid hope of treatment for Duchenne muscular dystrophy, but still gigantic financing needs to offer it on a larger scale: the Telethon is “a pivotal moment”while will be launched Friday November 29 from 6:45 p.m., until Saturday evening, its 38^e edition.

Presented by Sophie Davant, Nagui and Cyril Féraud, this edition is sponsored by the singer Mika. It is the young Sacha, 8 years old, who was diagnosed with Duchenne muscular dystrophy when he was 2 years old and who is today treated as part of gene therapy, who is the ambassador.

Packed with more than 23,000 events and challenges throughout France, the Telethon takes place a little earlier than usual, the first weekend in December being devoted to the reopening of Notre-Dame de Paris. Donations can be made in three forms: by calling 3637, during an event across the territory or on Telethon.fr.

“Pivotal moment”

This year is special, Serge Braun, scientific director of the French Association Against Myopathy (AFM), explained to Agence France-Presse, because “we finally have a treatment that could change the course of Duchenne muscular dystrophy, a disease previously thought to be incurable”. This rare disease, which destroys muscles and affects one in 3,000 boys in France, is the emblematic cause of the Telethon: in 1958 it was at the origin of the creation of the AFM, the association which organizes the event to fund research into genetic diseases.

Five children aged 6 to 10 benefited from a pioneering drug that allowed them to rebuild muscle, without significant side effects. Presented at two medical conferences, this 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 Serge Braun.

“We are at a pivotal moment: because we have these great results in research on gene therapy for Duchenne muscular dystrophy, but also because the resources we need to continue this great adventure are considerable”explains Laurence Tiennot-Herment, president of the AFM. The AFM-Téléthon also supports “around forty trials in humans concerning 33 different pathologies (…), but when we move on to human trials, we are looking at a cost of tens, or even hundreds of millions of euros”she specifies.

Another genetic disease where research has led to a pioneering drug: spinal muscular atrophy, a “quite dramatic neuromuscular disease, because there is paralysis of the muscles and death of the child around the age of 1 and a half or 2 years”underlines Professor Didier Lacombe, coordinator of the New Aquitaine neonatal screening center.

Diagnosed a few days after their birth, two one-year-old twins, Marley and Mylane, were able to benefit from it and are today “in good health”according to testimonies from their parents, one of the ambassador families of Telethon 2024. Until then, “we screened these babies when they had symptoms, around 6 to 9 months, at that time we injected them but we arrived too late: we had bedridden children”expose Didier Lacombe.

“The idea is to detect spinal muscular atrophy from birth: we fought with the AFM-Téléthon to set up this pilot program in France, we came up against quite complicated ethical and political debates, and we arrived there at the beginning of 2023 in two regions: the Grand-Est and Nouvelle-Aquitaine”he relates. This pilot program, deployed in 81 maternity wards, made it possible to screen 11 babies, eight of whom received gene therapy – the others could not be treated given the specific form of their disease.

“With a single injection – certainly expensive, but much less than long-term care – we are supposed to cure these children, it is the first real miracle of genomic medicine”praises Didier Lacombe, pleading for screening “generalized throughout the territory from 2025”. In France, around fifteen genetic diseases are detected at birth.