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
Ultragenyx Pharmaceutical announced the publication of the 96-week data from its Phase 3 trial of GENGLYCOS (DTX401), an AAV gene therapy for glycogen storage disease type Ia (GSDIa). The study showed a significant 61% mean reduction in daily cornstarch intake while maintaining glycemic control, including elimination of nighttime dosing in a substa…