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Feature|Articles|December 27, 2025 (Updated: January 2, 2026)
Assessing Antisense Oligonucleotide Apazunersen in Pediatric Angelman Syndrome: The Aspire Study
Author(s)Isabella Ciccone, MPH
Fact checked by: Kelly King
Apazunersen, an investigational intrathecal therapy, is being evaluated in the phase 3 Aspire trial for pediatric Angelman syndrome, with study completion anticipated in the second half of 2026.
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Welcome to NeurologyLive®'s Clinical Trial in Focus. Every month, an ongoing clinical trial in the landscape of neurology is featured, highlighting the design of the study, the targeted patient population, the enrollment criteria, the primary and secondary end points, and its potential implications for clinical care. This month’s spotlight is the global phase 3 Aspire study (NCT06617429) assessing apazunersen (Ultragenyx Pharmaceutical), an investigational antisense oligonucleotide therapy, in pediatric patients with Angelman syndrome (AS).1
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