Phase III Aspire Study Misses Both Efficacy Endpoints in Angelman Syndrome

Company: Ultragenyx · Drug: Apazunersen (Intrathecal ASO) · Trial: Aspire (Phase III) · Indication: Angelman Syndrome · Date: September 2, 2026 Summary Ultragenyx said the Phase III Aspire trial of intrathecal apazunersen in Angelman syndrome missed its primary Bayley-4 cognition endpoint and key secondary Multidomain…