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September 15, 2024 marks the 4th International Myotonic Dystrophies Awareness Day. This event, supported by over 60 organizations worldwide, aims to raise awareness among the general public, healthcare professionals and political decision-makers of the need to improve diagnosis, care and support for patients, and to speed up the development of medicines. The AFM-Telethon, which brings together French patients affected by these diseases, is committed to speeding up research. This new edition focuses on two specific areas in particular: raising awareness and educating clinical care teams, and preparing participants for clinical trials.

World Duchenne Muscular Dystrophy Day provides an opportunity to note the acceleration in the development of innovative therapies for this neuromuscular disease, which is the most common in children. Gene therapy is one of the most promising avenues. Genethon, the AFM-Telethon laboratory, is currently developing a drug candidate which has demonstrated its efficacy in the first patients to be treated. Discover our video of patient testimonials ! 

On Friday 31 August, 56 people from the Danish association MuskelsVindfonden, the Danish equivalent of AFM-Téléthon, visited the Institut de Myologie.

The laboratory of AFM-Telethon celebrates its 30th anniversary: watch the video series recounting the saga of this one-of-a-kind laboratory, which put France at the forefront of genome exploration, and has supported gene therapy through thick and thin, bringing it to its first successes today. Let us have a look back at this ongoing medical revolution.

They are #SoStrong! Families who fight on a daily basis against the disease, researchers who turn genes into medicine, volunteers, donors and partners who, every year, brave the cold… Thanks to them joining forces for the past 30 years, a medical revolution is underway today! Together, let’s keep on giving children and patients the strength to heal!

The World Duchenne Awareness Day takes place on September 7th, 2020! After many years of research, hopes for treatments become reality, specifically for Duchenne Muscular Dystrophy, one of Telethon’s most emblematic diseases, which was at the root of parents’ fight and of the creation of AFM-Telethon by the association’s pioneers. A gene therapy trial, stemming from research conducted in Genethon, a Telethon laboratory, should start in France and more broadly in the United Kingdom, Israel and the United States.

Last December 6th and 7th, people all across France rallied in favour of the 2019 Telethon, with donations totalling 74.5 million euros by the end of the television broadcast. Today, a few months later, we are very proud to announce the final result of our 2019 fundraising campaign: 87 026 262 euros. You are great!

It is with great sorrow that we share the sad news of the death of a great researcher, a pioneer of gene therapy and a fellow traveler of the AFM-Telethon. Jon A Wolff died on April 17, 2020 of esophageal cancer, the first signs of which he had detected during a visit to France 18 months earlier.

From September 12 to 17th, AFM-Telethon will hold two successive scientific congresses gathering close to 1000 international experts on myology and mitochondrial medicine at the Nice Acropolis Convention Centre. Registrations are open!

Through the collective mobilization of donors, volunteers, families, and partners, all united in solidarity to fight against rare diseases, the final result of the 2021 Telethon amounts to €85.933.166. Thank you!

During four days, from 12 to 15th of September, researchers, clinicians and myology experts have attended the seventh International Congress of Myology, organized by AFM-Téléthon, in Nice, France. A good opportunity to exchange and share knowledge about myology and innovative therapies. Let’s have a look back at Myology 2022 with our researchers’ video interviews.

"Advances in Charcot-Marie-Tooth (CMT) disease" is a new document, published by the French Muscular Dystrophy Association (AFM-Téléthon), which can be read and/or downloaded here.

The Téléthon 30-hour-long broadcast ended with donations totaling 78 051 091 euros. From Guebwiller to Cassis, Dijon, Lorient and Cap-Ferret, millions of people came together again for the Téléthon, this unique celebration with its thousands of events. A vital support for the families and researchers, “these saving lives maniacs” as Kev Adams called them. But while researchers’ victories multiply, all week-end long patients and their families reminded everyone of the urgency of finding new treatments. 

The AFM-Telethon sends you its best wishes for the New Year and wishes you a year 2023 full of color and joy!

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), has launched a pivotal clinical trial in Europe for treatment of Crigler-Najjar syndrome, a life threatening liver disease, in France, Italy and the Netherlands.