ANGELMAN SYNDROME
Clinical trials for ANGELMAN SYNDROME explained in plain language.
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Breakthrough treatment shows promise for rare genetic disorder
Disease control ENROLLING_BY_INVITATIONThis study continues testing GTX-102, an experimental treatment for Angelman Syndrome, to understand its long-term safety and effectiveness. It's open only to people who completed earlier GTX-102 trials. Researchers will monitor side effects and track developmental progress over …
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Mar 30, 2026 14:35 UTC
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Potential breakthrough treatment tested for rare genetic disorder
Disease control OngoingThis study is testing whether an investigational drug called GTX-102 can improve thinking skills and other symptoms in children with Angelman syndrome, a rare genetic disorder that affects development. Researchers are comparing the drug against a sham (placebo-like) procedure in …
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Mar 30, 2026 14:35 UTC