FDA raises concerns over Sarepta's Duchenne gene therapy
TL;DR Summary
FDA reviewers have raised concerns about the efficacy and safety of Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy, stating that the clinical studies conducted so far do not provide unambiguous evidence that the therapy is likely beneficial for ambulatory patients with the disease. The briefing documents were released ahead of an advisory committee hearing on Friday.
Topics:business#duchenne-muscular-dystrophy#fda#gene-therapy#healthcare#patient-safety#sarepta-therapeutics
- FDA reviewers had string of concerns about Sarepta's gene therapy for Duchenne, documents show STAT
- FDA reviewers flag concerns over Sarepta's gene therapy for muscle-wasting disorder Reuters
- 'It's transformational work,' Sarepta working on groundbreaking gene therapies in Columbus ABC6OnYourSide.com
- Sarepta's Duchenne gene therapy faces FDA advisory committee STAT
- Ahead Of Sarepta Advisory Committee, FDA Feels Congressional Pressure About Public Participation Pink Sheet
- View Full Coverage on Google News
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