Clinical Trial Update: Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

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Summary

Completed study reporting long-term safety and efficacy data for a gene therapy in Glycogen storage disease type I (GSD Ia) with results posted.

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Diseases / indications

• Glycogen Storage Disease Type

Drugs mentioned

• DTX401

Snippet

Sponsor: Ultragenyx Pharmaceutical Inc | Conditions: Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia) | Status: COMPLETED | Results posted: 2026-03-18