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🔬NCT07157254Phase 2RECRUITING

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

Study Summary

The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.

Conditions
Angelman Syndrome
Interventions / Drugs
No interventionGTX-102
View on ClinicalTrials.gov ↗
Sponsor
RARE
Ultragenyx Pharmaceutical
Rare Disease
C63/100
Study Details
Enrollment60 participants
Primary Completion—
Lead SponsorUltragenyx Pharmaceutical Inc
Data Retrieved2026-04-11 04:25:35.045702+00:00
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