Angelman Syndrome
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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 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 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 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.
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CLINICAL_TRIALS trial_result · 2016-11-21Clinical Trial Update: Study on Tolerability of Levodopa/Carbidopa in Children With Angelman Syndrome [Phase 1]
A Phase 1 study evaluated the tolerability of levodopa/carbidopa in children with Angelman syndrome. The trial has completed, and results we…
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CLINICAL_TRIALS trial_result · 2016-02-02Clinical Trial Update: Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW
This clinical trial update reports the completion of a study examining sleep abnormalities in individuals with Rett syndrome, Angelman syndr…
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CLINICAL_TRIALS trial_result · 2015-10-21Clinical Trial Update: Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome [Phase 2]
A Phase 2 clinical trial evaluated the efficacy and safety of minocycline for Angelman syndrome and has completed; no results are provided i…
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CLINICAL_TRIALS trial_result · 2012-09-24Clinical Trial Update: Dietary Supplements for the Treatment of Angelman Syndrome [NA]
A clinical trial evaluating dietary supplements for Angelman syndrome completed, with results posted in 2012.