Clinical Trial Update: A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) [Phase 3]

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Summary

Phase 3 study of AAV8-mediated glucose-6-phosphatase gene transfer for Glycogen Storage Disease Type Ia has completed.

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Diseases / indications

• Glycogen Storage Disease Type Ia

Drugs mentioned

• AAV8-G6PC gene therapy (Adeno-associated virus serotype 8-mediated glucose-6-phosphatase gene transfer)

Snippet

Sponsor: Ultragenyx Pharmaceutical Inc | Conditions: Glycogen Storage Disease Type IA | Phase 3 | Status: COMPLETED