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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.

Last December 4th and 5th, the 2020 Telethon took place in a difficult context due to the COVID19 crisis with donations totalling nearly 58.3 million euros by the end of the TV broadcast. A few months later, we are extremely proud to share the exceptional final result of our 2020 fundraising campaign: 77 298 024 euros. Thank you so much!

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.

On February 28, International Rare Disease Day will put the spotlight on the rare diseases community throughout the world. At this occasion, AFM-Telethon revisits thirty years of pioneering research and innovation carried out by its laboratory, Genethon, which has developed a high-level expertise in researching and developing preclinical and clinical gene therapy treatments for rare diseases.

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.

At the end of the television broadcast, the counter of the 2020 Telethon shows 58 290 120 euros. Despite the health crisis, which led to the cancellation of a very large number of events in towns and villages, the French people responded positively to this extraordinary Telethon: the one presenting the greatest victories of its history. Behind these victories, there are faces, first names, children, parents, changed lives, regained motions, smiles and laughter, and life who wins.

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!

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.

Enabling early detection and diagnosis of the disease is the aim of the new European Alliance for Newborn Screening in Spinal Muscular Atrophy (SMA NBS Alliance), launched on August 31st by SMA Europe, of which AFM-Telethon is a member.

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.