All the
latest

September 02, 2026

Ultragenyx Announces Phase 3 Aspire results in Angelman Syndrome

Phase 3 Aspire did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI) NOVATO, Calif., Sept. 02, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc.

Read more
September 01, 2026

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% Complete elimination of nighttime cornstarch dosing observed in 33% of DTX401

Read more