FDA approves therapy for GSDIa
The one-time gene therapy is intended to restore the deficient enzyme needed to release stored glucose during fasting.
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By
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Kathryn Wighton
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August 20, 2026
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The FDA granted accelerated approval to pariglasgene brecaparvovec-opnr (Genglycos) for glycogen storage disease type Ia.
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This therapy aims to reduce daily cornstarch intake in patients aged 8 years and older as part of nutritional management.
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Pariglasgene brecaparvovec-opnr is a gene therapy designed to deliver a functional glucose-6-phosphatase gene to the liver.
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Clinical trials showed a 31% mean reduction in daily cornstarch intake among treated patients compared to placebo.
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Serious adverse reactions include anaphylaxis and adrenal insufficiency, with warnings included in the prescribing information.
