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Generics Bulletin

Medtech Insight

Gene Therapies Without Randomized Clinical Trials: Marks Outlines Rare Disease Development Path

Nov 29 2023

• By Derrick Gingery

clinical trial and laboratory — Gene therapies in pediatric rare diseases may not need a randomized clinical trial to gain accelerated approval. • Source: Shutterstock

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Related Companies

Sarepta Therapeutics, Inc.

Additional Topics

United States Gene Therapy Rare Diseases Drug Approval Standards Clinical Trials Regulation Pediatrics Biologics Review Pathway BioPharmaceutical

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