Glycogen Storage Disease Type Ia (GSDIa) research hub

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adenoassociated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia.

This study looks at a new gene therapy called Pariglasgene brecaparvovec for people with a rare liver condition called GSDIa. It aims to see if this new treatment can help reduce the amount of cornstarch they need to eat daily to keep their blood sugar stable.