ANGELMAN SYNDROME
Clinical trials for ANGELMAN SYNDROME explained in plain language.
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New spinal injection therapy tested in children with rare genetic disorder
Disease control CompletedThis early-stage clinical trial tested the safety and tolerability of an experimental drug called GTX-102 in children with Angelman syndrome. The drug was given as multiple injections into the spinal fluid. The main goal was to see how well children handled the treatment and to m…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Apr 03, 2026 14:42 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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National registry launched to map angelman syndrome in italy
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 data to better understand the common medical and behavioral challenges associated wi…
Matched conditions: ANGELMAN SYNDROME
Sponsor: FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS • Aim: Knowledge-focused
Last updated Apr 03, 2026 14:43 UTC