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It is a positive commitment, but France must set an example by perpetuating and amplifying a truly ambitious national policy.

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.

On 16 December 2021, the UN adopted the first-ever UN Resolution on “Addressing the Challenges of Persons Living with a Rare Disease and their Families.” A huge recognition for families with rare diseases.

The 2021 Telethon ended with donations totaling 73 622 019 euros. An amount reflecting the generosity of the general public, the energy felt during thousands of Telethon events throughout France and abroad, and the strength of families’ fight and researchers’ determination. This unique combination is the cause for a true revolution in medicine that saves lives and opens up new prospects for treatment.

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.

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!