Condition

Glycogen Storage Disease Type Ia

Treatments and trials tagged Glycogen Storage Disease Type Ia, with the same country and access zoom as New Developments.

All New Developments

Glycogen Storage Disease Type Ia breakthroughs

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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…

This is for people 8 years and older with glycogen storage disease type Ia who still rely on carefully timed…