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

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Summary

Ultragenyx announced publication of successful 96-week randomized, placebo-controlled trial results for GENGLYCOS (DTX401), an AAV gene therapy for glycogen storage disease type Ia, in The Journal of Inherited Metabolic Disease.

Clinical Trial Data

Primary Endpoint

Met

Outcome Details

96-week randomized, placebo-controlled trial with crossover treatment published in The Journal of Inherited Metabolic Disease

Importance:6/10

Sentiment:

0.70

AAV gene therapyGSDIarandomized controlled trialpublicationclinical data

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Published by yahoo_finance on September 1, 2026 8:05 PM

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