Angelman syndrome
MONDO:0007113A neurogenetic disorder characterized by severe intellectual deficit and distinct facial dysmorphic features.
Also known as: Angelman syndrome, Angelman’s syndrome, Angelman syndrome (Type 1), Angelman syndrome (Type 2), AS, Angelman syndrome chromosome region, happy puppet syndrome (formerly), happy puppet syndrome, formerly
16 clinical trials for this condition and its sub-types.
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Broader categories
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New angelman drug shows promise in early safety trial
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 a spinal injection. Researchers monitored side effect…
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC
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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 (a specific genetic type). The goal was to see how the drug moves through the body and if it is safe. Researchers also looked at brain activity changes to see if the drug works as expected. T…
Phase: PHASE2 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC
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New registry aims to shed light on angelman syndrome
Knowledge-focused CompletedThis completed registry study collected information from 82 people with Angelman syndrome in Italy. The goal was to track medical and behavioral problems linked to the condition. No treatments or interventions were tested; instead, the data will help researchers better understand…
Sponsor: FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:09 UTC