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Genethon and Children's Hospital Boston Get FDA approval for a Wiskott Aldrich Gene Therapy Trial
Date:7/21/2011

we are committed to utilizing state-of-the-art cell and molecular therapies to treat devastating pediatric diseases. The trial in WAS is particularly noteworthy as it represents a continuing transatlantic scientific and clinical research collaborative effort in gene therapy and huge multi-disciplinary team effort at Children's," comments Dr. David A. Williams, Sponsor-Investigator of study.

About Genethon

Genethon is a not-for-profit research centre that was created by the French Muscular Dystrophy Association (AFM) and funded almost exclusively by donations from France's annual Telethon. Its goal is to deliver gene therapies to patients with rare diseases. With over 200 scientists, physicians, engineers and regulatory affairs specialists, Genethon is one of the world's leading centers for preclinical and clinical research and development in the field of gene therapy. Genethon also has a biomanufacturing platform for clinical-grade vectors and is involved in building the world's largest facility for pre-industrial pilot production (Genethon BioProd), which will open in Evry in 2012.

www.genethon.fr

About the AFM

The French Muscular Dystrophy Association (AFM) federates patients with neuromuscular diseases (genetic diseases that kill, muscle after muscle) and their parents. Thanks in great part to donations from France's annual Telethon (90 million euros in 2010), the AFM has become a major player in biomedical research into rare diseases in France and worldwide. It is currently funding 36 clinical trials on 30 different genetic diseases affecting the eyes, the blood, the brain, the immune system, the muscle... Thanks to its Genethon research lab, the AFM stands out through its unique ability to produce and test its own gene-based medicines.

More information at www.afm-telethon.fr

About Children's Ho
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SOURCE Children's Hospital Boston
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