Overview

Long-term Extension of GTX-102 in Angelman Syndrome

Status:
ENROLLING_BY_INVITATION
Trial end date:
2029-02-01
Target enrollment:
Participant gender:
Summary
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Phase:
PHASE3
Details
Lead Sponsor:
Ultragenyx Pharmaceutical Inc