Telethon: research is made possible by you!

The first edition of the telethon was in 1987. How do you view what has been accomplished since then?

Philippe Legrand : A look of hope! I have been involved with the AFM-Telethon for 29 years, but above all I am a father. My son Brice suffered from Duchenne muscular dystrophy. We learned about the disease when he was 7 years old, it was in 1995. We were told: Your son has a muscle disease. At the time, research into myology (part of anatomy that concerns muscles) and rare diseases was in its infancy. We knew that for Brice, there would be no treatment. And today, during this 2024 edition, you will see little Sacha. He is the same age as Brice was. Thanks to the AFM-Téléthon, he was able to benefit from a clinical trial when he was 2 years old. He walks, he even climbs trees! There it is, the journey taken.

We couldn't wait for others to find solutions. So the AFM-Téléthon created them, thanks to the generosity of donors. Genethon, for example, the pioneering laboratory of the AFM-Téléthon, has established itself as one of the leaders in gene therapy for rare diseases. This is where revolutionary treatments are being developed that save the lives of children suffering from spinal muscular atrophy, but also diseases of the immune system, blood, liver, vision, etc.

What is the current research focused on?

P.L. : Curing neuromuscular diseases and rare, progressive diseases is the priority mission of the AFM-Téléthon, which funds more than 350 research programs each year. The benefits of this research are also valid for more common diseases, such as heart failure, eye diseases, etc. Current research is focused on stem cells, to develop new treatments, and also on neonatal screening.

For example, it was previously impossible in France to detect spinal muscular atrophy before birth. It is a rare, often fatal genetic disease that causes progressive muscle atrophy. Without treatment, 95% of children with the most serious form die before the age of 2. In 2022, AFM-Téléthon launched Depisma, a screening experiment in 81 maternity hospitals in the Grand Est and Nouvelle Aquitaine regions. Thanks to innovative therapies available, it is possible to effectively treat babies before symptoms even appear. This experiment was a success and the High Authority of Health authorized the generalization of systematic screening throughout the territory. It's a giant step.

Is this why donations remain essential?

P.L. : Let it be clear: if there had not been so many donations over all these years, science would not have progressed so quickly. You make research possible. And there are so many victories left to be won! Today, there are 7,000 rare diseases, 80% of which are genetic. And we only have treatment for 5% of them.

And then the AFM-Téléthon is not just about research: it is also about supporting patients and their families. Facilitating access to diagnosis and best care practices, allowing as many people as possible to participate in the increasing number of clinical trials, is one of the association's priorities. Just because you have a progressive illness doesn't mean you can't live your life as best as possible. My son Brice, despite his myopathy, studied and obtained his doctorate in marine biology. He obtained it posthumously, because he died a few weeks before his defense, 8 years ago. Like the seabirds he loved so much, he was freedom-loving and always amazed by life. We must not give up, for our young people.

Will you be in Avesnes-sur-Helpe on November 29 and 30?

P.L. : Obviously ! It has been 12 years since a northern city was an ambassador for the Telethon and the Northern Department is co-organizing this event. Live on France Télévisions, there will be 30 hours of challenges, including that of Stéphane Plancke, suffering from girdle muscular dystrophy. He will cycle 200 kilometers from Bergues to join us in Avesnes! We are all mobilized. The town of Avesnes has put a special prize pool online, complementary to that of 36 37. It will remain open until February. We're counting on you!