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Angelman Syndrome clinical trials at UCSD

3 in progress, 2 open to eligible people

Showing trials for
  • GTX-102 in Pediatric Subjects with AS

    open to eligible people ages 4-17

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

    San Diego, California and other locations

  • 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.

    San Diego, California and other locations

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

    Sorry, accepting new patients by invitation only

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

    San Diego, California and other locations

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