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This year, the Telethon – the charity event organized by the French Association against myopathies (AFM) – takes place on December 2 and 3. But what are the latest advances? We take stock.
The 36th edition of the Telethon 2022 will take place this year on France 2 in the company of Kev Adams, Sophie Davant, Nagui, Cyril Féraud as well as the families of the patients, the scientists and the teams of France Televisions. The opportunity to encourage the French to donate, to help all children with rare genetic diseases (Crigler-Najjar syndrome, spinal muscular atrophy, myotubular myopathy, Leber optic neuropathy, etc.) that were previously thought to be incurable.
11 diseases saw advances in 2022
Thanks to this solidarity event, many treatments and therapeutic avenues have emerged, particularly around gene therapy – a therapeutic strategy that consists of introducing genes into a person’s cells or tissues.
Nearly 1,800 gene therapy trials are in progress around the world.and they are inspired by the pioneering research that has been carried out thanks to the Telethon”confides Serge Braun, scientific director of the French Association against myopathies.
“We feel a lot of pride when we see the children around us treated and a lot of humility: 95% of diseases remain to be treated even if gene therapy has become widespread“, specifies Frédéric Revah, the general manager of the Telethon.
According to the AFM-Téléthon association, in total, 11 neuromuscular diseases have made progress over the past year:
- SMN1-related proximal spinal muscular atrophy;
- myotonic dystrophy type 1 (Steinert’s disease);
- myotonic dystrophy type 2;
- congenital muscular dystrophies;
- Duchenne and Becker muscular dystrophies;
- Charcot-Marie-Tooth disease;
- autoimmune myasthenia gravis;
- facioscapulohumeral myopathy;
- congenital myopathies;
- girdle muscular dystrophy;
- inflammatory myopathies.
Doctissimo is proud to be a partner of the Telethon again this year. Thanks to your donations, the AFM-Tééthon is accelerating research to provide patients with innovative medicines. Find out what this donation will cost after tax reduction.
