- RecruitingPHASE2
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
This study is looking at a new treatment called GTX-102 for Angelman Syndrome. It aims to see if the treatment is safe and if it helps improve the condition in people of different ages and genetic types. Some people will get the treatment, and some won't at first.
For: Angelman SyndromeUnited States · Argentina · Brazil - Enrolling by invitationPHASE3
Long-term Extension of GTX-102 in Angelman Syndrome
This study is looking into the long-term safety and effects of a treatment called GTX-102 for people with Angelman Syndrome. It's for those who have already been in an earlier study with this treatment.
For: Angelman SyndromeUnited States · Australia · Canada