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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!

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.

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

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.

Their determination and expertise are at the origin of great scientific advances and major victories. Thanks to the researchers in the Telethon laboratories, children suffering from formerly incurable diseases have been saved. 

Results published in The New England Journal of Medicine demonstrate the safety and tolerance for gene therapy in Crigler-Najjar syndrome as well as its efficacy at the highest dose. This rare liver disease is characterized by a toxic build-up of bilirubin in the body, which can cause significant neurological damage and become fatal. 

In an article published in July 2023, an Italian researcher traces the birth and development of myology. This medical science of muscle and its diseases has developed over time, not only thanks to the advent of tools and technologies that improve our knowledge of muscle, but also thanks to the international collaborations that have marked its history.

The 10th World Duchenne Awareness Day, on September 7, is a call to action to break down the physical, social and healthcare barriers faced by the 250,000 people around the world affected by this disease. It's also an opportunity to take stock of advances in research into this disease, which is emblematic of our Association. 

The 30th of September is Limb-Girdle Muscular Dystrophy Awareness Day. At this occasion, the LGMD Interest Group of the AFM-Téléthon is planning an information day about these diseases in Evry. 

You live abroad and want to play a part in the Telethon? Join the Telethon for French people abroad and help us break records of mobilization around the world! 

A review published by two researchers at I-Stem, one of the AFM-Téléthon laboratories, takes stock of human pluripotent stem cells (hiPS) and confirms their main advantages in neuromuscular diseases: improving understanding and identifying new treatments.

The first clinical results of the gene therapy for the treatment of limb-girdle muscular dystrophy type 2I/R9 show good preliminary efficacy and biological tolerability. These first results have been presented during oral communications during the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) and will be presented in the 2023 International Limb-Girdle Muscular Dystrophy Conference in Washington DC.