Angelman Syndrome
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome [Phase 3]
Ionis Pharmaceuticals reports a Phase 3 trial (REVEAL) of ION582 for Angelman syndrome, currently recruiting.
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) [Phase 2]
Phase 2 clinical trial update for GTX-102 in Angelman Syndrome, currently recruiting.
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: A Natural History Study of Angelman Syndrome
Massachusetts General Hospital is conducting a natural history study of Angelman syndrome and is currently recruiting participants. The stud…
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CLINICAL_TRIALS research · 2026-03-16Clinical Trial Update: A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome [Phase 1, Phase 2]
A Phase 1/2 study evaluating MVX-220 for safety and efficacy in Angelman syndrome is currently recruiting.
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CLINICAL_TRIALS research · 2026-03-16Clinical Trial Update: Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome
A clinical trial is exploring eye-tracking to study social perception abnormalities in children with Angelman Syndrome and is currently recr…
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CLINICAL_TRIALS research · 2026-02-27Clinical Trial Update: Italian Angelman Syndrome Registry
Italian Angelman Syndrome Registry study has completed, as indicated by the completion status in ClinicalTrials.gov.
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: IDMet (RaDiCo Cohort) (RaDiCo-IDMet)
The RaDiCo-IDMet cohort study sponsored by INSERM is enrolling participants to study imprinting-related disorders including Silver-Russell s…
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CLINICAL_TRIALS research · 2026-02-04Clinical Trial Update: Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) [Phase 3]
Phase 3 trial update for GTX-102 in pediatric Angelman syndrome; the study is currently active but not recruiting.
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CLINICAL_TRIALS research · 2026-01-23Clinical Trial Update: Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) [NA]
Rush University Medical Center is conducting a web-based intervention (WINGS) for parents of youths with several genetic syndromes, and the …
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CLINICAL_TRIALS research · 2025-11-14Clinical Trial Update: HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome [Phase 1, Phase 2]
This document describes a Phase 1/2 clinical trial evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple a…
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CLINICAL_TRIALS research · 2025-08-22Clinical Trial Update: Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome
Expanded access/compassionate use program for Rugonersen in Angelman Syndrome is available.
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CLINICAL_TRIALS research · 2025-07-31Clinical Trial Update: Parent and Infant Inter(X)Action Intervention (PIXI) [NA]
Clinical trial update for the PIXI Inter(X)Action Intervention (NCT03836300), currently enrolling by invitation across Fragile X Syndrome, A…
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CLINICAL_TRIALS research · 2025-05-20Clinical Trial Update: Natural History Study for Patients With Angelman Syndrome
A natural history study for Angelman Syndrome is currently recruiting to observe the progression of the condition over time.
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CLINICAL_TRIALS research · 2025-04-03Clinical Trial Update: Angelman Syndrome Natural History Study
This is a natural history study at Boston Children's Hospital recruiting to characterize the progression of Angelman Syndrome.