Angelman syndrome due to a point mutation Advocacy Hub | UniteRare

At a Glance

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11Recruiting trials

Regulatory Watchboard

10 events

Mar 2026A Natural History Study of Angelman Syndrome

Massachusetts General Hospital

TrialRECRUITING

Oct 2025A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome

MavriX Bio, LLC — PHASE1, PHASE2

TrialRECRUITING

Oct 2025A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

Ultragenyx Pharmaceutical Inc — PHASE2

TrialRECRUITING

Feb 2025Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome

Assistance Publique - Hôpitaux de Paris

TrialRECRUITING

Dec 2024Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

Ultragenyx Pharmaceutical Inc — PHASE3

TrialACTIVE NOT RECRUITING

Jul 2024Long-term Extension of GTX-102 in Angelman Syndrome

Ultragenyx Pharmaceutical Inc — PHASE3

TrialENROLLING BY INVITATION

Dec 2023Angelman Natural History Study - FAST Spain

Puerta de Hierro University Hospital

TrialRECRUITING

Mar 2022Study of the Prevalence of Autistic Traits in Angelman Syndrome

IRCCS Eugenio Medea

TrialRECRUITING

Dec 2021HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

Ionis Pharmaceuticals, Inc. — PHASE1, PHASE2

TrialRECRUITING

Oct 2021Natural History Study for Patients With Angelman Syndrome

Centre Hospitalier Universitaire de Liege

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

Total programs

No financial assistance programs currently listed for Angelman syndrome due to a point mutation.
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Clinical Trial Landscape

11 active trials

2Phase 3

1Phase 2

6Unknown

2PHASE1, PHASE2

11Total recruiting

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Recent News & Research

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Specialist Network

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