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
19 clinical trials for this condition and its sub-types, 19 tagged with Angelman syndrome itself.
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Browse by category →Sub-types of Angelman syndrome
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New hope for angelman syndrome: experimental drug enters Mid-Stage trial
Disease control OngoingThis study tests an experimental drug called GTX-102 (apazunersen) in 60 people with Angelman syndrome, a genetic disorder causing developmental delays and seizures. Participants range from 1 to 65 years old and are grouped by age and genetic type. The goal is to see if the drug …
Phase 2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Sep 11, 2026 00:00 UTC
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New drug aims to control angelman syndrome long-term
Disease control OngoingThis phase 3 trial tests the long-term safety of GTX-102 (apazunersen) in 255 people with Angelman syndrome, a genetic disorder causing developmental delays and seizures. Participants have already completed an earlier GTX-102 study and will continue receiving the drug. The main g…
Phase 3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Sep 11, 2026 00:00 UTC
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New hope for angelman syndrome: experimental drug ION582 enters human trials
Disease control OngoingThis study tests a drug called ION582 for people with Angelman syndrome, a genetic condition causing developmental delays and seizures. The drug is given via a spinal injection. The main goal is to check safety and how the body processes the drug. Up to 70 participants aged 0 to …
Phase 1/2 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 22, 2026 00:00 UTC
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New drug hopes to boost brain function in kids with rare genetic disorder
Disease control OngoingThis Phase 3 trial tests a drug called GTX-102 (apazunersen) in 129 children with Angelman syndrome, a genetic condition causing severe developmental delays. The study compares the drug to a sham procedure to see if it improves cognitive function and other symptoms over about 11 …
Phase 3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Aug 08, 2026 00:03 UTC
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Can early parent coaching help infants with rare genetic disorders thrive?
Disease control By invitation onlyThis study tests a program called PIXI that coaches parents of infants diagnosed with rare neurogenetic disorders (such as Fragile X, Angelman, or Down syndrome) during the first year of life. The program combines education about the disorder, guided parent-child interaction acti…
Sponsor: RTI International • Aim: Disease control
Last updated Jul 15, 2026 00:00 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
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Can a video-based therapy tame tough behaviors in kids with rare genetic disorders?
Symptom relief OngoingThis study tests a virtual behavioral therapy (Functional Behavioral Training) for children aged 2-12 with genetic syndromes like Fragile X, Angelman, or Rett syndrome who have challenging behaviors. The therapy teaches parents how to identify what triggers problem behaviors and …
Sponsor: Rush University Medical Center • Aim: Symptom relief
Last updated Sep 20, 2026 00:00 UTC