FDA ApprovedUnited States
First FDA-Approved Gene Therapy for Glycogen Storage Disease Type Ia
GENGLYCOS (pariglasgene brecaparvovec-opnr) — one-time gene therapy to help lower daily cornstarch needs in glycogen storage disease type Ia (ages 8 and older)
FDA accelerated approval on 19 August 2026 for GENGLYCOS (pariglasgene brecaparvovec-opnr) suspension — a one-time AAV8 gene therapy given by intravenous…
This is for people 8 years and older with glycogen storage disease type Ia who still rely on carefully timed cornstarch and may be candidates for labelled US…