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

Status:
RECRUITING

Trial end date:
2030-01-01

Target enrollment:

Participant gender:

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

PHASE2

Ultragenyx Pharmaceutical Inc