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Read more about AFM-Telethon’s social missions, main activities, and key figures in this new edition of Cure through Innovation.

Myology Institute's team has developed and tested a new approach to gene therapy, a so-called “decoy” approach, for Steinert’s disease or myotonic dystrophy type 1.

Genethon announced that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.

Families, researchers, volunteers, partners… all are ready to organize the 35th Telethon! A crucial event for patients and their family, and for the whole of medicine.

This booklet features AFM-Telethon’s main missions, actions and key figures.

« Advances in Steinert’s disease » and « Advances in myotonic dystrophy type 2 » are two new documents, published by the French Muscular Dystrophy Association (AFM-Téléthon), which can be read and/or downloaded here.

Preliminary results from the European gene therapy trial for Crigler-Najjar syndrome, conducted by Généthon in collaboration with European network CureCN, were presented at the EASL (European Association for the Study of the Liver) annual International Liver Congress on June 26. Based on initial observations, the drug candidate is well tolerated and the first therapeutic effects have been demonstrated, to be confirmed as the trial continues.

A first participant was dosed at I-Motion, the pediatric clinical trial platform for neuromuscular diseases located at Trousseau hospital in Paris, as part of the gene therapy trial in Duchenne muscular dystrophy (DMD) conducted by Genethon.

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.

Généthon, a unique scientific and human adventure, turns 30 this year. Created by a patient organization and funded by the Téléthon, this laboratory has put France at the forefront of genome exploration and of the gene therapy revolution.

What is AFM-Telethon? What are its missions? What were the highlights of 2019? The answer is in the Cure through Innovation booklet.

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.