Ultragenyx Doses First Patient in Aurora Study for Angelman Syndrome Treatment

Ultragenyx Pharmaceutical has initiated the Aurora study, evaluating the efficacy and safety of GTX-102 (apazunersen) for Angelman syndrome. The study aims to expand the treatment population to include younger and older patients and those with non-deletion AS genotypes not included in the Phase 3 Aspire study. The fully enrolled Phase 3 Aspire study is focused on patients aged 4 to 17 years with a confirmed full maternal UBE3A gene deletion.