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

▤ Summary

Ultragenyx announced publication of 96-week Phase 3 data for GENGLYCOS, also known as DTX401, in glycogen storage disease type Ia. The study showed continued reductions in daily cornstarch use while patients maintained glycemic control and improved fasting tolerance. Many participants also reduced or eliminated nighttime cornstarch dosing, which lowered treatment burden. Ultragenyx said the therapy remained generally well tolerated and supported by an acceptable safety profile. The company also noted that GENGLYCOS has already received U.S. FDA approval for eligible patients.

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