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Phase 3
Development Phase
1
Upcoming Catalysts
0
Historical Events
Regulatory Approvals
GTX-102 Phase 3 Results Expected
GTX-102 • Angelman Syndrome
Target Indication
Angelman Syndrome
Clinical Trial
Last updated: 12/4/2025
RARE
Ultragenyx Pharmaceutical Inc.
OTC Deficiency
MPS IIIA
Hereditary Inclusion Body Myopathy
MPS 7
Osteogenesis Imperfecta