Angelman Syndrome clinical trials at UCSD
3 in progress, 2 open to eligible people
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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