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W19-209-1
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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)
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Dr. Rebecca Riba-Wolman
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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.
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Glycogen Storage Disease Type Ia (GSDIa)
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N/A
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Although this study is not actively recruiting and enrollment was by invitation only (subjects had to of been enrolled in the 401GSDIa01 Study) if you have any questions about this study please contact Shaylee King at PediatricClinicalTrials@uchc.edu or phone number 860-679-6584.
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