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🔬NCT06415344Phase 3ENROLLING BY INVITATION

Long-term Extension of GTX-102 in Angelman Syndrome

Study Summary

The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

Conditions
Angelman Syndrome
Interventions / Drugs
GTX-102
View on ClinicalTrials.gov ↗
Sponsor
RARE
Ultragenyx Pharmaceutical
Rare Disease
C63/100
Study Details
Enrollment255 participants
Primary Completion—
Lead SponsorUltragenyx Pharmaceutical Inc
Data Retrieved2026-04-11 04:25:35.045729+00:00
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