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Sarepta, FDA And The Dangers Of Strong Early Results

Executive Summary

The small Duchenne patient community is already convinced eteplirsen works, but FDA doesn't seem to agree and the advisory committee didn't seem to offer a way out except through a controlled trial.

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Attention to the departure of neurology reviewer Ron Farkas from FDA has focused on what it means for Sarepta’s eteplirsen application, but there are larger regulatory environment issues that deserve more scrutiny.

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