Ultragenyx Pharmaceutical Inc. announced that the first patient has been dosed in the Aurora study, which is evaluating the efficacy and safety of GTX-102 (apazunersen), an investigational treatment for Angelman syndrome. The Aurora study will enroll approximately 60 participants aged 1 to under 65 years old with Angelman syndrome across all genotypes, expanding the population studied beyond the previous Phase 3 Aspire trial. The Aurora study features four cohorts based on age and genotype, with varying primary endpoints, and includes both single-arm and randomized groups. The Aspire Phase 3 study, focused on patients aged 4 to 17 years with full maternal UBE3A gene deletion, has completed enrollment, with results expected in the second half of 2026. No new research results have been presented yet; current announcements pertain to trial enrollment and study initiation.