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Pariglasgene Brecaparvovec-opnr Approved for Glycogen Storage Disease 1a
FDA granted accelerated approval to pariglasgene brecaparvovec-opnr, a one-time liver-directed AAV8 gene therapy, based on a 31% reduction in cornstarch intake versus placebo.
- Published by
- CGTLive
- Evidence type
- Regulatory
FDA granted accelerated approval to pariglasgene brecaparvovec-opnr, a one-time liver-directed AAV8 gene therapy, based on a 31% reduction in cornstarch intake versus placebo.
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