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Exondys 51’s Development: Was Placebo-Controlled Trial Possible?

 
• By Derrick Gingery

Sarepta says US FDA said ‘no,’ but agency repeatedly urged the company to pursue the approach.

Sarepta Therapeutics Inc. and FDA appeared at odds over whether a placebo-controlled trial should have, or could have, been conducted as part of the later development stages of Exondys 51 in Duchenne muscular dystrophy.

A Sarepta official said recently that the company, patients and physicians concluded that a Phase III placebo-controlled trial would not be appropriate given the promising results seen in previous

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