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NOVATO, Calif., Nov. 09, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its ...

The data presented at FAST reinforce that the Aspire Phase 3 primary endpoint of cognition, as measured by Bayley-4, appears very well powered to show statistically significant separation between the ...

Phase 1/2 data show improvements across all domains and confirm that Phase 3 Aspire study is amply powered to establish efficacy of GTX-102 Phase ... Therapeutics (FAST) Global Science Summit ...

GTX-102 has been granted Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation from the FDA and Orphan Designation and PRIME designation from the EMA. About the ...