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Pariglasgene brecaparvovec clinical trials
2 indexed studies · 0 currently recruiting
- Ongoing, recruitingPhase I and Phase II (Integrated)- Other
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.
For: Glycogen storage disease type IaSpain · Netherlands - Ongoing, recruitingTherapeutic confirmatory (Phase III)
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.
For: Glycogen Storage Disease Type Ia (GSDIa)Denmark · Netherlands · Spain