Pariglasgene brecaparvovec clinical trials

A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease Type Ia (GSDIa)

This study looks at a new gene treatment called Pariglasgene brecaparvovec for adults with a rare liver condition called Glycogen Storage Disease Type Ia (GSDIa). It aims to see how safe it is and if it helps control blood sugar levels long-term.