Press releases
  Edgar Gomes, Inserm researcher in the Group MYOLOGY at the Institute of Myology, in collaboration with researchers at the Sloan-Kettering Institute (New York) has identified several proteins involved in “correctly” positioning nuclei, which is necessary for muscles to function. Their results are published in a letter in the Nature review, dated March 18. Read the complete press release - 2 pages - 112 ko
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Gene therapy: Encouraging results in a Phase I clinical trial in limb-girdle muscular dystrophy type 2C - 12/01/2012The results of a Phase I clinical trial of gene therapy for limb-girdle muscular dystrophy type 2C (a rare neuromuscular disease) have just been published in the journal Brain on January 11, 2012. The trial started in December 2006 and has been sponsored by Généthon (the not-for-profit research lab created by the French Muscular Dystrophy Association (AFM) and which is funded almost exclusively by donations from France's annual Telethon). The trial at Pitié-Salpêtriere (AP-HP) is being led by principal investigators Professor Serge Herson (Head of the Department of Internal Medicine 1) and Professor Olivier Benveniste (Institute of Myology). The study's primary objective was to evaluate the safety of local injection of increasing doses of an adeno-associated virus (AAV) vector harboring a "healthy" copy of the gene for gamma-sarcoglycan (the defective protein in this disease). Secondary objectives included the assessment of local and systemic immune reactions and the quality of gene transfer in the injected muscles in terms of efficacy, expression and distribution.
Access the complete press release - 4 pages - 920 Ko
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The counter of the 25th edition of the Telethon finished at 86,119,425 euros! The extraordinary popular enthusiasm of the Telethon, which makes this event unique in the world, overcame a particularly difficult economic and social context. Proof once again that the strong bond that unites the population to the Telethon is stronger than ever. Proof that with you, everything becomes possible!
Today, thanks to all of you, we are approaching the critical stage of clinical trials and the first treatments. The ultimate goal that we set together 25 years ago is close at hand. A new decade of outstanding advances is starting, thanks to you! Access the complete press release - 1 page - 244 ko
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Partnership for a gene therapy Clinical trial signed between Généthon and Children’s Hospital Boston - 05/01/2011A unique multicenter international study of gene therapy for a rare genetic disease (Wiskott-Aldrich syndrome) led in London, Paris and Boston.
Généthon, the not-for-profit biotherapy laboratory funded by the French Muscular Dystrophy Association (AFM) thanks to the donations collected during the French Telethon, and Children's Hospital Boston announce that they have initiated a partnership to conduct a gene therapy clinical trial for Wiskott-Aldrich Syndrome (WAS). This rare genetic condition is a life-threatening immunodeficiency disease. Genethon is sponsoring parallel trials at Great Ormond Street Hospital in London and Hopital Necker-Enfants Malades in Paris (Press release – Feb 24, 2010) and will be supplying the vector for the trial at the US site. The vector is a latest-generation lentiviral vector that incorporates several safety features designed to avoid the complications caused by early-generation vectors. The vector is manufactured by Genethon in Evry. Altogether, the WAS gene therapy trials in London, Paris and Boston will constitute a unique multicenter collaboration to accelerate the testing of new advanced therapies for rare conditions. Access the complete press release - 3 pages - 272 ko
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The French Muscular Dystrophy Association (AFM) is organizing the 4th International Congress of Myologie, which will be held from the 9th of May until the 13th of May in the Lille Grand Palais. During these five days, close to 1,000 participants coming from the five continents will be informed and updated on the scientific and medical advances made in the realm of muscle science and related disorders. |
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Tomorrow, around thirty innovative biotherapy and genetic eye disease experts* will gather together at Généthon (Evry) to review, for the first time, the studies carried out so far as part of the Réseau Thérapie Génique Oculaire (R-TGO – Ocular Gene Therapy Network). The network was launched in 2009 by Généthon and is co-led by José-Alain Sahel (Institut de la Vision, Paris), Fabienne Rolling (Nantes) and Laurence Tiennot-Herment (Généthon/Association Française contre les Myopathies). Made up of project teams, representatives from centres of excellence in the field of eye diseases and gene therapy experts from the Généthon laboratory, its objective is to accelerate the launch of gene therapy trials for genetic eye diseases in the next five years. Access the complete press release - 2 pages - 18 ko
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New therapies for degenerative diseases - 28/05/2010Paris, May 6th, 2010. Several research groups have allied with industry partners to launch EndoStem, a large European collaborative project to advance the treatment of muscle diseases that affect a large number of patients. The project will promote the development of strategies involving skeletal muscle, blood vessels, immune system and stem cells to repair damaged muscle tissue directly. Access the complete press release - 1 page - 35 ko
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FINAL TELETHON 2009 TOTAL: 95,200,117 EUROS - 20/04/2010To mark the announcement of the final total raised by Telethon 2009, AFM IS HOLDING AN OPEN DAY at 11 research laboratories in France €95, 200,117: the total raised by the 2009 23rd Telethon organised on 4-5 December by AFM and France Télévisions, with the support of radio stations from the Radio France group. The scale of this total in difficult economic times bears witness to the general public’s commitment to the concepts of solidarity and pushing one’s own boundaries promoted by AFM. It also represents the concerted efforts of one million donors, 200,000 volunteers and 5 million people taking part in almost 20,000 events, ranging from the most traditional to the outright crazy, in over 10,000 French towns and villages. Access the complete press release - 1 page - 43 ko
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Généthon, the not-for-profit biotherapy laboratory operated by the French Muscular Dystrophy Association (AFM) with funding from the country's annual Telethon, has just obtained approval from the French and British health authorities for a Phase I/II clinical trial of gene therapy for a rare immunodeficiency, Wiskott-Aldrich syndrome (WAS). The Généthon-sponsored trial will be performed both in France (led by Professors Alain Fischer (1) and Marina Cavazzana-Calvo (2) at Necker Children's Hospital, Paris) and in the UK (led by Professor Adrian Thrasher at London's Great Ormond Street Hospital). With just a few days to go to Rare Disease Day 2010 (February 28th, with a focus on "Bridging Patients and Researchers”), initiation of this trial marks a new step towards curing rare diseases with innovative biotherapies. Access the complete press release - 2 pages - 56 ko
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Frédéric Revah brings over 20 years' experience in the pharmaceutical and biotech industries and academic research to the job. Généthon will notably benefit from his acknowledged expertise in the management of therapeutic innovation. "Today, Généthon is one of the world's benchmark labs for the development of innovative therapies for rare diseases, thanks to a unique, synergistic set of skills (ranging from research and clinical development to large-scale biomanufacturing) and exceptional support from the AFM and its donors. My goal is to pursue and accelerate this progress and deliver gene therapies to patients with rare diseases in general and neuromuscular diseases in particular", stated Généthon's new Chief Executive. Access the complete press release - 2 pages - 48 ko
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