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 in treatment and crossover groups reduced daily cornstarch intake by a mean 61% while maintaining glycemic control. Most participants achieved at least a 50% reduction, suggesting the approach can materially cut cornstarch intake without worsening glucose outcomes.