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Illustration for article: On december 3 and 4 : let's go for Telethon 2021!

On december 3 and 4 : let's go for Telethon 2021!

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.
Illustration for article: Telethon 2020: 58 290 120 euros!

Telethon 2020: 58 290 120 euros!

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.
Illustration for article: The Généthon laboratory, gene therapy pioneer, is 30 years old!

The Généthon laboratory, gene therapy pioneer, is 30 years old!

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.
Illustration for article: Cure through Innovation 2020 is available online!

Cure through Innovation 2020 is available online!

What is AFM-Telethon? What are its missions? What were the highlights of 2019? The answer is in the Cure through Innovation booklet.
Illustration for article: A European Alliance for Newborn Screening in Spinal Muscular Atrophy

A European Alliance for Newborn Screening in Spinal Muscular Atrophy

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.
Illustration for article: Myositis: A new classification system based on phenotypic, biological and immunological criteria

Myositis: A new classification system based on phenotypic, biological and immunological criteria

Illustration for article: France revives positive European Momentum against Rare Diseases

France revives positive European Momentum against Rare Diseases

It is a positive commitment, but France must set an example by perpetuating and amplifying a truly ambitious national policy.
Illustration for article: A new approach to gene therapy for Steinert’s disease

A new approach to gene therapy for Steinert’s disease

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
Illustration for article: Genethon’s Lentiviral Vector-Based Gene Therapy Demonstrates Long-Term Safety and Efficacy for Wiskott-Aldrich Syndrome

Genethon’s Lentiviral Vector-Based Gene Therapy Demonstrates Long-Term Safety and Efficacy for Wiskott-Aldrich Syndrome

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.
Illustration for article: First-ever United Nations resolution to increase visibility for persons living with a rare disease and their families

First-ever United Nations resolution to increase visibility for persons living with a rare disease and their families

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.
Illustration for article: Myology 2022 and MitoNice 2022: registration and abstract submission are open

Myology 2022 and MitoNice 2022: registration and abstract submission are open

From September 12 to 17, 2022, AFM-Telethon will hold two international scientific congresses at the Nice Acropolis Convention Centre. You can submit your abstracts until April 30, 2022
Illustration for article: € 73 622 019 thank you! The fight can continue!

€ 73 622 019 thank you! The fight can continue!

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.
Illustration for article: The Cure through Innovation booklet is available

The Cure through Innovation booklet is available

This booklet features AFM-Telethon’s main missions, actions and key figures.
Illustration for article:  Advances in research : two new documents are available

Advances in research : two new documents are available

« 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.
Illustration for article: Preliminary results of clinical trial for rare liver disease Crigler-Najjar syndrome presented at EASL congress

Preliminary results of clinical trial for rare liver disease Crigler-Najjar syndrome presented at EASL congress

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.
Illustration for article: 77 298 024 € raised for the 2020 Téléthon!

77 298 024 € raised for the 2020 Téléthon!

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!
Illustration for article: First Patient dosed in Clinical Trial of Investigational Gene therapy for Duchenne Muscular Dystrophy

First Patient dosed in Clinical Trial of Investigational Gene therapy for Duchenne Muscular Dystrophy

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.
Illustration for article: Genethon, a unique strike force against rare diseases

Genethon, a unique strike force against rare diseases

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.
Illustration for article: Thirty years of Genethon: the saga in videos

Thirty years of Genethon: the saga in videos

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.
Illustration for article: On december 4 and 5, the force of the Téléthon will be with you

On december 4 and 5, the force of the Téléthon will be with you

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!