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The Telethon 2023 ended on a high of €80,671,222, which shows the confidence and exceptional mobilization of donors and volunteers for this year's event, rich in victories against disease and promises for all those waiting for the diagnosis and treatment that will change their lives. We must not give up. 

Finding treatments for neuromuscular diseases by combining artificial intelligence, stem cells and pharmacological screening is the aim of the new DREAMS research consortium, coordinated by I-Stem. An unprecedented project!

A gene therapy developed at Genethon by Ana Buj Bello's team is proving effective in a rare and very severe muscle disease: myotubular myopathy. Review of the results published online in The Lancet Neurology on November 15. 

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.

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 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. 

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. 

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.

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. 

The Genother biocluster, dedicated to accelerating the development of gene therapy drugs, of which Genethon is one of the founders, is one of the 5 winners of the France 2030 call for expressions of interest. This labelling, announced on 16 May by the French President, will strengthen France's leadership in the field of gene therapy, one of the most dynamic areas of biomedical research.

ILTOO Pharma and the European consortium MIROCALS have just announced the signing of a license agreement for the development of a low-dose Interleukin 2 as a potential treatment for amyotrophic lateral sclerosis (ALS). The AFM-Telethon helped finance this clinical study, which shows an improvement in patient survival. 

Thank you to everyone: donors, volunteers mobilized throughout France and abroad, families, partners, personalities! Thanks to your mobilization on December 2 and 3, the Telethon 2022 raised 90,839,067 euros! This tremendous result - the best since 2016 - shows how much the French people support the families’ fight against rare diseases.

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. 

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), has launched a pivotal clinical trial in Europe for treatment of Crigler-Najjar syndrome, a life threatening liver disease, in France, Italy and the Netherlands.