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1,000 researchers in schools: the third edition

300 schools, more than 31,000 pupils reachable in 2015 … In view of the success of the previous editions, the AFM-Telethon and APBG join together one more to bring the world of research in schools during November. Again this year, all over France, science teachers of life and earth (SVT) of colleges and high schools … [Read more]

Telethon 2015: 4 families, 4 stories, 4 fights against rare disease

Four families, all affected by rare diseases, will be the ambassadors of thousands, all gathered around the message: “The Fight of Parents, The Life of Children.” On December 4th and 5th, together with Marc Lavoine, the official ambassador of Telethon 2015, four families will represent the fight of the AFM-Telethon.  Séthi, Léo, Nicolas and Marie … [Read more]

World premiere : differentiation of pluripotent stem cells to muscle fiber

This major breakthrough in neuromuscular diseases has just been published August 3, 2015 in Nature Biotechnology. “With our method, which does not require any genetic manipulation, we obtain muscle fibers which contract, but also satellite stem cells “ Olivier Pourquié, IGBMC and Harvard University (photo credit : Lola Vélasquez/IGBMC) More information with the original article  … [Read more]

A new trial in gene therapy led by UCLA in partnership with Genethon

10 children with an immunodeficiency disorder, commonly referred to as X-linked CGD, will be enrolled to receive a stem cell gene therapy, due to Dr Kohn’s team and Genethon. Genethon, AFM-Téléthon’s laboratory research, engineered the virus delivery system designed to transport the gene therapy into patients’ blood-forming stem cells. Genethon is sponsoring a parallel European … [Read more]

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]