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ASGCT 18 th Annual Meeting 2015: Ana Buj Bello receives the Outstanding New Investigator Award

This award recognizes four researchers every year for the quality and importance of their work in gene and cell therapy. Ana Buj Bello is awarded for 10 years of pioneering work on gene therapy and currently leads a translational gene therapy program for myotubular myopathy, a very severe muscular disease t due to mutations in … [Read more]

Effectiveness of innovative gene therapy treatment demonstrated in canine model of Duchenne muscular dystrophy

A collaboration involving three laboratories supported by the AFM-Telethon, Atlantic Gene Therapies (AFM-Telethon, Inserm UMR 1089, Université de Nantes,Nantes University Hospital), Généthon (Evry) and the Institute of Myology (Paris), demonstrated the effectiveness of an innovative gene therapy treatment in the canine model of Duchenne muscular dystrophy. This work, published in the journal Molecular Therapy in … [Read more]

Discover Telethon 2014 exceptional program

An exceptional sponsor, 4 families, 30 hours of broadcast on France Télévisions and 2,000 volunteers in the starting blocks: Telethon 2014 promises a lot of surprises! The AFM-Telethon, France Télévisions and Garou, sponsor of Telethon 2014 invited everyone on december 5th and 6th, on France Télévisions channels and throughout France, for a spectacular new edition! … [Read more]

European Medicines Agency recommends first-in-class medicine for treatment of Duchenne muscular dystrophy

The European Medicines Agency’s Committee for Medicinal Products for Human Use(CHMP) has recommended granting a conditional marketing authorisation for Translarna (ataluren), an orphan-designated medicine for the treatment of Duchenne muscular dystrophy caused by nonsense mutations. Translarna is to be used in patients aged five years and older who are able to walk… Read the press … [Read more]

Genosafe celebrates its 10th anniversary

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]

Development for a Treatment for Myotubular Myopathy

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]

Efficacy of gene therapy demonstrated in canine and murine models of myotubular myopathy

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]

Telethon 2013: 5 families, 5 stories, 5 fights against muscle disease

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]

Seed capital fund: an incentive to treat rare diseases

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]

Drisapersen: significant improvement in walking distance in patients with Duchenne muscular dystroph

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]