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Ultragenyx Completes Patient Enrollment in Angelman Syndrome Phase 3 Trial
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Ultragenyx Completes Patient Enrollment in Angelman Syndrome Phase 3 Trial
Aug 1, 2025 2:35 AM

05:11 AM EDT, 08/01/2025 (MT Newswires) -- Ultragenyx Pharmaceutical ( RARE ) said late Thursday it completed patient enrollment in a phase 3 trial of GTX-102 for the treatment of Angelman syndrome, a neurogenetic disorder.

The trial, which enrolled 129 subjects, is expected to be completed in H2 2026.

The company said it expects to start a separate study of the drug in additional stages and genotypes in H2 2025.

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