RARE ▲ +2.0% - Ultragenyx Pharmaceutical Inc. $RARE - 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 rose 2.0% following the publication of positive 96-week trial data for its GENGLYCOS gene therapy in glycogen storage disease type Ia, which demonstrated efficacy in a randomized, placebo-controlled study with crossover treatment. The peer-reviewed results in The Journal of Inherited Metabolic Disease validate the therapeutic approach for this rare metabolic disorder and support the drug's clinical profile. 01-SEP-2026 04:06 PM ET