ANGELMAN SYNDROME
Clinical trials for ANGELMAN SYNDROME explained in plain language.
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New drug trial hopes to ease angelman syndrome symptoms in kids
Disease control CompletedThis study tested a drug called alogabat in 48 children aged 5-17 with Angelman syndrome caused by a specific gene deletion. The goal was to see how the drug moves through the body and if it is safe, while also checking for early signs it might help control symptoms. The study la…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE2 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated May 17, 2026 05:47 UTC
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New drug trial aims to tame angelman syndrome in kids
Disease control CompletedThis study tested a new medicine called GTX-102 in 74 children with Angelman Syndrome, a rare genetic disorder causing developmental delays and seizures. The main goal was to see if the drug is safe and tolerable when given as multiple injections into the spine. Researchers monit…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 14, 2026 12:04 UTC
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No treatment, just data: italian registry maps angelman syndrome challenges
Knowledge-focused CompletedThis study created a national registry for people with Angelman syndrome in Italy. Researchers collected information on medical and behavioral problems from 82 participants. No experimental treatments were given; the goal was to better understand the condition and its common issu…
Matched conditions: ANGELMAN SYNDROME
Sponsor: FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS • Aim: Knowledge-focused
Last updated May 11, 2026 20:41 UTC