Core Insights - Ultragenyx Pharmaceutical Inc. has fully enrolled the Phase 3 Aspire study for GTX-102, targeting Angelman Syndrome, with approximately 129 participants aged 4 to 17 [1][2] - The study aims to evaluate the efficacy of GTX-102 in improving cognitive function and other key areas over a 48-week period, with completion expected in the second half of 2026 [2][3] Company Overview - Ultragenyx is a biopharmaceutical company focused on developing therapies for rare and ultra-rare genetic diseases, with a diverse portfolio aimed at addressing high unmet medical needs [7][8] - The company has received multiple designations from the FDA and EMA for GTX-102, including Breakthrough Therapy Designation and Orphan Drug Designation, indicating its potential significance in treating Angelman Syndrome [3] Product Details - GTX-102 (apazunersen) is an investigational antisense oligonucleotide therapy designed to reactivate the expression of the UBE3A gene, which is crucial for addressing the underlying cause of Angelman Syndrome [3][4] - The Phase 3 Aspire study involves a randomized design where participants receive either GTX-102 or a sham comparator, with the primary endpoint focused on cognitive improvement [2][3] Disease Context - Angelman Syndrome is a rare neurogenetic disorder affecting approximately 60,000 individuals in commercially accessible regions, characterized by cognitive and motor impairments, seizures, and the need for continuous care [4][5] - There are currently no approved therapies for Angelman Syndrome, highlighting the urgency and potential impact of GTX-102 in the treatment landscape [6]