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By physician referral or invitation only

Ultragenyx

The purpose of this study is to evaluate if the drug GTX-102 is effective with the Angelman population. This is a Phase 3 study, that is a double-blind, sham-controlled study.

Age & Gender

  • 4 years ~ 17 years
  • Male, Female, Gender Inclusive

Contact the Team

Location

Thank you for your interest, but this study is recruiting by invitation only.

United States (Nationwide)

Additional Study Information

Principal Investigator

Elizabeth Jalazo
Carolina Institute for Developmental Disabilities

Study Type

Clinical or Medical
Interventional

Study Topics

Genetics and Genetic Disorders

IRB Number

24-2478

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