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Clinical Trials: Glycogen Storage Disease Type Ia (GSDIa)
IRB No. W19-209-1 (Dr. Rebecca Riba-Wolman, PI): 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 serves to continue to evaluate the safety and efficacy of the AAV8 Mediated Gene Therapy in those subjects infused with it in the 401GSDIa01 Study, this study is approximately 4 years in length. There are no investigative products given in this study.