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Find here all the latest news on AFM-Telethon and rare diseases.

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Xavier Nissan, chercheur à Istem
Research
01/12/2023

I-Stem, coordinator of an innovative research consortium

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!

équipe Isabelle Richard à Généthon
Research
27/10/2023

Limb-Girdle Muscular Dystrophy Type 2I/R9: first gene therapy results 

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.

« Immunologie et Thérapie Génique des Maladies du Foie » de Généthon, dirigée par le Dr Giuseppe Ronzitti
Research
21/08/2023

Crigler-Najjar: gene therapy shows its effectiveness

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. 

ADN Helice
Research
22/05/2023

Genother Biocluster: Accelerating the development of gene therapy drugs in France 

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.

best-of research
Research
23/01/2023

Protagonists of a medical revolution, researchers testify 

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. 

document advance in research
Research
07/12/2022

Advances in research: a new document is available

"Advances in Charcot-Marie-Tooth (CMT) disease" is a new document, published by the French Muscular Dystrophy Association (AFM-Téléthon), which can be read and/or downloaded here.

Myology 2022 congress
Research
19/09/2022

A look back at Myology 2022 

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.

Téléthon 2018 Victoire Crigler-Najjar
Research
02/07/2021

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.