News

European leader in providing services  for the evaluation of gene therapy products, GenoSafe team specializes in the evaluation of cellular and gene therapy products (gene therapy products, vaccines, etc …) Wide spectrum expertise GenoSafe offers its expertise in various stages, from proof of concept to the placing on the market of a product and works … [Read more]

Promising preclinical data recently published in Science Translational Medicine, Audentes Therapeutics Inc. and Genethon announce that they have entered into an agreement to develop AT001 for the treatment of Myotubular Myopathy A significant step forward “Our agreement with Genethon is a significant step forward towards the development of a treatment for patients with this serious, … [Read more]

A team of French researchers, led by Dr. Anna Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, have demonstrated the efficacy of gene therapy in models of myotubular myopathy, an extremely severe neuromuscular disease in children. Transfer of the MTM1 gene, which is deficient in the … [Read more]

On December 6th and 7th, together with Patrick Bruel, the official ambassador of Telethon 2013, five families will represent the fight of the AFM-Telethon. Sandrine, Luke, Vanessa, Emmanuelle and Geraldine and their children live throughout France. Despite different family situations and histories, they all have one thing in common: the fight against the disease that … [Read more]

With the Caisse des Dépôts Enterprises (a management company in charge of promoting equity capital financing of growing French SMEs in the technological sector or in traditional industry which create value and jobs) and the Fonds National d’Amorçage, FNA (a fund set up by the French government to provide capital to companies developing innovative technologies … [Read more]

Yesterday, during The Cold Spring Harbor International Conference in China, GSK announced a statistically significant improvement in walking distance in Duchenne muscular dystrophy patients treated with drisapersen for one year. Drisapersen, which was developed by Prosensa then GSK, is a synthetic antisense oligonucleotide that skips exon 51 of the dystrophin gene. The initial development of … [Read more]

The 6th International Rare Disease Day, associated with the AFM Telethon, will be held February 28th 2013 in France and in over 60 countries around the world. Around this time, the global community of rare diseases will be rallying to bring into the spotlight, rare diseases and the millions of people they touch. Rare diseases … [Read more]

An American study conducted in collaboration with Genethon has provided proof-of-concept for enzyme replacement therapy in mice. The treatment carried out in mice involved replacing the missing myotubularin in this disease by a laboratory-modified form of myotubularin that is capable of specifically targeting the inside of skeletal muscle cells. Intramuscular injections of this product improved … [Read more]

The counter of Telethon 2012 stopped at 81,065,239€. But your solidarity must not stop now. This year’s Telethon will remain the challenge of all challenges: the challenge of donors and volunteers in the face of crisis, the challenge of researchers and families in the face of disease … Together, we have tackled them, with determination and … [Read more]

A few days before the launch of the Telethon on December 7th and 8th , the AFM-Telethon organised a press conference in Paris, presenting the Association’s challenges for 2013 and the researchers that it supports. Laurence Tiennot-Herment, President of the AFM-Telethon, recalls the challenge of the coming years: “To be the first consistent non-profit stakeholder in … [Read more]