Ignore the bickering between shareholders of Sarepta Therapeutics (NASDAQ: SRPT) and GlaxoSmithKline (NYSE: GSK) /Prosensa (NASDAQ: RNA) for a moment, because these are exciting times for the roughly 1 in 4,000 newborns worldwide who are born with Duchenne muscular dystrophy. There are two drugmakers currently knocking on the doorstep of the Food and Drug Administration, each with a unique drug -- eteplirsen for Sarepta, and drisapersen for Glaxo/Prosensa -- that has the ability to slow or stop the muscle-destroying effects of DMD. With very few treatment options currently available to DMD patients, and an average lifespan of just 25 years, this is very encouraging news. Until recently, the data on the two drugs was similarly positive, but the edge in recent weeks had been sliding back into drisapersen's favor. This big concern that has plagued Sarepta shareholders recently is that the FDA has had a difficult time accepting increased dystrophin production as a primary endpoint to improved results in DMD patients. It also hasn't helped Sarepta's case that its study is only mid-stage in nature and based on just 12 patients. This was the primary reason the FDA refused to accept an accelerated new drug application filing from Sarepta. But, … Continue reading
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