ANGELMAN SYNDROME
Clinical trials for ANGELMAN SYNDROME explained in plain language.
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Hope for angelman syndrome: new drug targets cognitive function in kids
Disease control OngoingThis study tests an experimental drug called GTX-102 in 129 children with Angelman syndrome caused by a specific gene deletion. The goal is to see if it improves cognitive function, such as thinking and learning, over about 11 months. Children receive either the drug or a sham (f…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 26, 2026 11:24 UTC
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New hope for angelman syndrome: Long-Term drug safety trial begins
Disease control ENROLLING_BY_INVITATIONThis study looks at the long-term safety of a drug called GTX-102 for people with Angelman Syndrome, a genetic disorder that causes developmental delays and seizures. About 255 participants who were in earlier GTX-102 trials will continue taking the drug to see if it remains safe…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 22, 2026 14:02 UTC