GlobeNewswire Clinical StudiesOriginal article
Ultragenyx Announces the Publication of a Successful 96-Week Randomized, Placebo-Controlled Trial with Crossover Treatment of GENGLYCOS™ (also known as DTX401) AAV Gene Therapy in GSDIa in The Journal of Inherited Metabolic Disease
Data ReadoutpositiveGENGLYCOSPositive
AI Analysis
Summary
Ultragenyx announced publication of successful 96-week randomized, placebo-controlled trial results of GENGLYCOS (DTX401) gene therapy in GSDIa, showing a mean 61% reduction in daily cornstarch intake while maintaining glycemic control. The positive trial results were published in The Journal of Inherited Metabolic Disease.
Clinical Trial Data
Primary Endpoint
Met
Outcome Details
96-week randomized, placebo-controlled trial with crossover showed mean reduction in daily cornstarch intake of 61% at Week 96 while maintaining glycemic control, with most participants achieving at least 50% reduction
Importance:6/10
Sentiment:
0.85
gene therapyAAVGSDIaclinical trialpublication
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Published by GlobeNewswire Clinical Studies on September 1, 2026 8:05 PM