25 April 2016 - The agency sits on a new treatment for a deadly muscular disease.

No government agency controls the fate of more people than the Food and Drug Administration, which has the power to deny children a treatment that could help them walk. The FDA is reviewing an experimental drug for muscular dystrophy, and the outcome could determine the quality of life for thousands—and whether companies continue to invest in curing rare diseases.

On Monday an FDA advisory committee will consider eteplirsen, a drug by Boston-based Sarepta designed to treat a strain of Duchenne muscular dystrophy, which is a genetic disorder that weakens every muscle in the body. The condition usually affects boys, who by age 12 or so can no longer walk, and over time damages the heart and lungs. The fatality rate is 100%, and most do not live past 25.

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