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News about "Ultragenyx"
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).
Ultragenyx | 04/09/2026 | By News Bureau | 132
FDA Grants Accelerated Approval to Ultragenyx's Genglycos for Rare GSDIa
Gene therapy becomes the first treatment designed to directly address the underlying cause of glycogen storage disease type Ia in patients aged eight and older.
Ultragenyx | 21/08/2026 | By News Bureau | 102
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