1. Pink Sheet

Sarepta Protocol Changes Could Impact Future Duchenne Trials

 
• By Sue Sutter

If US FDA Commissioner Gottlieb allows use of in-dwelling ports in ESSENCE study, Office of Pediatric Therapeutics will assess whether the determination could serve as precedent for placebo-controlled trials of other exon-skipping compounds in Duchenne muscular dystrophy.

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The US FDA’s decision on amending a Sarepta Therapeutics Inc. trial protocol could make other placebo-controlled studies in Duchenne muscular dystrophy easier to conduct but is unlikely to extend beyond this limited therapeutic setting.

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