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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. Charcot-Marie-Tooth (CMT) disease is a group of clinically and genetically heterogeneous diseases, characterized by damage to the peripheral nerves of the upper and lower limbs. Read more

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 … [Read more]

Human induced pluripotent stem cells, a valuable tool for better understanding and treating neuromuscular diseases

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. Read the news

World Duchenne Awareness Day: all united !

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. … [Read more]

Crigler-Najjar: gene therapy shows its effectiveness

« Immunologie et Thérapie Génique des Maladies du Foie » de Généthon, dirigée par le Dr Giuseppe Ronzitti 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 … [Read more]

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, … [Read more]

90,839,067 euros for the Telethon 2022: we have not given up. Thank you!

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. “On December 2 and … [Read more]

Muscles, a major public health issue

Because muscles are a major public health issue, AFM-Telethon and the Institute of Myology, an international center of expertise on muscles and their diseases, are carrying out a project for a Myology Foundation. In addition, to raise awareness on myology among the public, they are launching two events dedicated to this public health issue in … [Read more]

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. This … [Read more]

Duchenne Muscular Dystrophy : First child dosed in Clinical Trial of Investigational Gene therapy

A first patient suffering from Duchenne muscular dystrophy received a first dose of the investigational gene therapy GNT0004 in an international phase I/II/III multicener trial for which Genethon is the sponsor. More information on the gene therapy clinical trial lead by Genethon.