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Promising Long-term Outcomes for DTX401 AAV Gene Therapy in Glycogen Storage Disease Type Ia Insights from Ultragenyx...
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Promising Long-term Outcomes for DTX401 AAV Gene Therapy in Glycogen Storage Disease Type Ia Insights from Ultragenyx...
Abstract Ultragenyx Pharmaceutical Inc. has reported positive longer-term data from a Phase 3 clinical study evaluating DTX401, an adeno-associated virus (AAV) gene therapy, for the treatment of Glycogen Storage Disease Type Ia (GSDIa). This article summarizes the outcomes observed at Week 96 of the study, highlighting significant reductions in dietary cornstarch intake while maintaining stable blood glucose levels and improved fasting tolerance…
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