FDA Advisory Committee Evaluates Sarepta's Gene Therapy for Duchenne Amid Concerns.
TL;DR Summary
The FDA advisory panel will meet on Friday to discuss Sarepta Therapeutics' experimental gene therapy for Duchenne muscular dystrophy. This will be the first hearing for a Duchenne drug from Sarepta since 2016, when the FDA overruled its committee's recommendations and authorized an earlier therapy from the company despite limited evidence, leading to celebrations from families and outcry from senior agency officials.
Topics:business#advisory-panel#duchenne-muscular-dystrophy#fda#gene-therapy#healthcare#sarepta-therapeutics
- What to know about a pivotal FDA hearing on Sarepta's gene therapy for Duchenne STAT
- 'It's transformational work,' Sarepta working on groundbreaking gene therapies in Columbus ABC6OnYourSide.com
- Ahead Of Sarepta Advisory Committee, FDA Feels Congressional Pressure About Public Participation Pink Sheet
- FDA reviewers had string of concerns about Sarepta's gene therapy for Duchenne, documents show STAT
- Sarepta's Duchenne gene therapy faces FDA advisory committee STAT
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