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GlobeNewswire·

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

At Week 96, participants across treatment and crossover groups experienced mean reduction in daily cornstarch intake of 61% while maintaining glycemic control, with most participants achieving reduction of at least 50%