Latest news

The initial results of Genethon's gene therapy trial for Duchenne muscular dystrophy were presented on Tuesday 23 April at the Myology 2024 congress in Paris.

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.

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.

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.

"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.

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.

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.