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Patients Can't Rescue Sarepta's Eteplirsen

This article was originally published in The Pink Sheet Daily

Executive Summary

Hours of testimony was clearly compelling, but advisory committee agreed with FDA concerns about the Duchenne muscular dystrophy drug's efficacy.

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US FDA Panel For Sarepta’s Gene Therapy Heavy On Temporary Voting Members

Only four of 14 voting panelists are regular members of the Cellular, Tissues and Gene Therapies Advisory Committee, with scheduling conflicts precluding participation by many others, the FDA said. Three of the temporary members are veterans of prior muscular dystrophy drug reviews.

The Greatest FDA Advisory Committees Of All Time Part IV: Neurology Lays Claim To The Crown

We look back at another of the greatest FDA advisory committees of the last 20 years. This time: the nearly 12-hour meeting in 2016 on Sarepta’s eteplirsen for Duchenne muscular dystrophy.

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