Sarepta’s DMD Gene Therapy, Like Exondys 51, Is Foundational, Advocates Argue
Executive Summary
The Duchenne muscular dystrophy candidate should be approved because it will help grow development in the space, patient groups argue, not just because of the value the gene therapy could provide to those receiving the treatment itself.
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Sarepta’s DMD Gene Therapy: Approval Delayed And Indication Narrowed, But Still On Track
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