Data as of Oct 5, 2026Based on 77 AI responses
Reviewed by Dimitry Apollonsky ·
GENGLYCOS is a prescription, one-time gene therapy (pariglasgene brecaparvovec-opnr) used alongside nutritional management to reduce daily cornstarch intake in adults and children aged 8 and older with glycogen storage disease type Ia (GSDIa). It is the first and only FDA-approved gene therapy for GSDIa, approved under accelerated approval based on reductions in daily cornstarch consumption, with continuation potentially contingent on demonstrated clinical benefit. Safety considerations include risk of allergic and infusion reactions, liver enzyme elevations requiring corticosteroids and liver monitoring, and adrenal gland issues, with guidance on vaccination, pregnancy, and contraception.
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