Tuesday, November 27, 2012 Finding a treatment for Duchenne muscular dystrophy (DMD) wont just mean developing one drug, says Chris Garabedian, president and CEO of Sarepta Therapeutics (Nasdaq:SRPT). It will mean developing as many as 20. The fatal inherited disorder affects one in 3,500 young boys, confining most of them to wheelchairs by the age of 12. Few patients with it live past the age of 30. Years of telethons by the Muscular Dystrophy Association and comedian Jerry Lewis have raised awareness of the disease, but today, available treatments are mostly limited to steroids - which only prolong the boys ability to walk by 2-5 years - and surgery, including heart transplants, according to the National Human Genome Institute. After Sareptas announcement this week that it will begin development of a fourth drug candidate for the treatment of DMD, Garabedian told Mass High Tech that the company is close to understanding the very origins of the disease in mutations of one of the longest genes in the human body, which is responsible for creating the muscle protein, dystrophin. Seraptas approach is to develop drugs which skip certain sections of the DNA where mutations inhibit production of dystrophin. While that wouldnt … Continue reading
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