Ultragenyx Doses First Patient in Aurora Study for Angelman Syndrome Treatment

Thursday, Oct 30, 2025 8:08 am ET1min read

Ultragenyx Pharmaceutical has dosed the first patient in the Aurora study evaluating GTX-102 for Angelman syndrome. The study will expand the treatment population to include younger and older patients, and those with other non-deletion genotypes not included in the Phase 3 Aspire study. The Aurora study will enroll approximately 60 participants across all genotypes, with some additional countries or regions included.

Ultragenyx Doses First Patient in Aurora Study for Angelman Syndrome Treatment

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