ANGELMAN SYNDROME
Clinical trials for ANGELMAN SYNDROME explained in plain language.
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Experimental injection tested in children with rare genetic disorder
Disease control CompletedThis study tested the safety of an experimental drug called GTX-102 in children with Angelman syndrome, a rare genetic disorder that affects development. Researchers gave the drug through spinal injections to 74 children to see how well they tolerated it and to measure drug level…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Mar 31, 2026 12:22 UTC
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Brain drug tested for rare genetic disorder in kids
Disease control CompletedThis study tested a drug called alogabat in children and teenagers with Angelman syndrome, a rare genetic disorder. The main goals were to see how the drug moves through the body, if it is safe, and if it changes brain wave patterns. Forty-eight participants aged 5 to 17 took the…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE2 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Mar 27, 2026 12:39 UTC
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Italian registry maps the Real-World impact of angelman syndrome
Knowledge-focused CompletedThis project created a national registry to collect health and behavioral information from people in Italy diagnosed with Angelman Syndrome. It did not test any treatments. The goal was to gather real-world data to better understand the common medical and behavioral challenges as…
Matched conditions: ANGELMAN SYNDROME
Sponsor: FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:30 UTC