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Angelman Syndrome clinical trials at University of California Health

4 in progress, 1 open to eligible people

Showing trials for
  • Angelman Syndrome Natural History Study

    open to all eligible people

    The goal of this study is to conduct a prospective, longitudinal natural history study of children and adults with Angelman Syndrome using investigator-observed and parent-reported outcome measures to obtain data that will be useful for future clinical trials.

    at UCSD

  • GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

    Sorry, in progress, not accepting new patients

    The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with deletion-type Angelman Syndrome (AS).

    at UCSD UCSF

  • Long-term Extension of GTX-102 in Angelman Syndrome

    Sorry, in progress, not accepting new patients

    The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

    at UCLA UCSD

  • REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome

    Sorry, in progress, not accepting new patients

    The purpose of this study is to evaluate the efficacy and safety of obudanersen in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.

    at UCLA

Our lead scientists for Angelman Syndrome research studies include .

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