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 trial_result · 2026-01-09Clinical Trial Update: A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome [Phase 1, Phase 2]
Ultragenyx reports the completed Phase 1/2 study evaluating the safety and tolerability of GTX-102 in children with Angelman syndrome. The t…
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CLINICAL_TRIALS trial_result · 2026-01-07Clinical Trial Update: Long-term Extension of GTX-102 in Angelman Syndrome [Phase 3]
This notice reports a long-term extension study for GTX-102 in Angelman Syndrome, a Phase 3 trial that is currently enrolling by invitation.
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CLINICAL_TRIALS trial_result · 2025-12-04Clinical Trial Update: A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype. [Phase 2]
Phase 2 study evaluating the pharmacokinetics, safety, and mechanism of action of alogabat in children and adolescents with Angelman syndrom…
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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 trial_result · 2025-08-24Clinical Trial Update: A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS) [Phase 1]
Phase 1 study evaluating safety, tolerability, pharmacokinetics and pharmacodynamics of RO7248824 in participants with Angelman Syndrome has…
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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 trial_result · 2025-07-18Clinical Trial Update: An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome [Phase 3]
Open-label Phase 3 study evaluating OV101 for Angelman syndrome was terminated, with results posted.
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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.
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CLINICAL_TRIALS trial_result · 2025-01-31Clinical Trial Update: An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome [Phase 2]
This update notes the completion of a Phase 2 open-label study evaluating the safety, tolerability, and pharmacokinetics of oral NNZ-2591 in…
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CLINICAL_TRIALS trial_result · 2024-01-08Clinical Trial Update: A Study of OV101 in Individuals With Angelman Syndrome (AS) [Phase 3]
Phase 3 trial of OV101 in Angelman Syndrome has been completed with results posted in 2024.
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CLINICAL_TRIALS trial_result · 2023-04-18Clinical Trial Update: Minocycline in the Treatment of Angelman Syndrome [NA]
Clinical trial update reporting completion and results posting for minocycline in Angelman Syndrome.
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CLINICAL_TRIALS trial_result · 2022-03-31Clinical Trial Update: A Study in Adults and Adolescents With Angelman Syndrome (STARS) [Phase 2]
This clinical trial update reports results from a Phase 2 study (STARS) in adults and adolescents with Angelman syndrome; the trial status i…
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CLINICAL_TRIALS trial_result · 2017-11-30Clinical Trial Update: Single Dose Pharmacokinetic (PK) Study [Phase 1]
Healx AI completed a Phase 1 single-dose pharmacokinetic study evaluating an investigational therapy for Angelman Syndrome and Fragile X Syn…
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CLINICAL_TRIALS trial_result · 2017-01-13Clinical Trial Update: A Trial of Levodopa in Angelman Syndrome [Phase 2, Phase 3]
A Phase 2/3 clinical trial evaluating levodopa in Angelman Syndrome has completed with results posted in 2017.