Ultragenyx reports positive Phase 1/2 results for GTX-102, targeting Angelman syndrome treatment.

Ultragenyx Pharmaceutical announced promising Phase 1/2 results for GTX-102, an antisense oligonucleotide aimed at treating Angelman syndrome, a rare neurogenetic disorder. Data indicate improvements in key cognitive areas, supporting the upcoming Phase 3 Aspire study, which plans to enroll around 120 patients. The study will focus on cognitive enhancements and has received multiple designations from regulatory bodies for its potential benefits.

November 09, 2024
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