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Data shows Cambridge biotech’s muscular dystrophy treatment is effective

10/07/2014 by


A Cambridge biotechnology company developing a treatment for a severe form of muscular dystrophy that progresses rapidly presented three years of data Thursday that show the drug is helping to slow the effects of the fatal, muscle-wasting disease.

With the positive results, Sarepta Therapeutics will seek approval from the Food and Drug Administration later this year with the goal of selling the drug, called eteplirsen, in 2015, to treat the disease known as Duchenne muscular dystrophy. If approved, it would be Sarepta’s first commercial drug.

“We’re very encouraged by this data,” Sarepta’s chief executive, Chris Garabedian, said in an interview. “This is more evidence that the FDA can look at to positively review eteplirsen’s treatment benefits.”

Duchenne muscular dystrophy occurs in about 1 of every 3,500 boys, destroying muscle function and quality of life over time. The disease moves swiftly, robbing most boys of the ability to walk by their teen years, and damaging their respiratory muscles. Duchenne patients typically do not live past their 20s.

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