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 deletion-type 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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