天使综合征药物研究进展汇总

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clinicaltrials

The Global Angelman Syndrome Registry
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2026-09-03
CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2026-08-24
Phase III Clinical Study of Rugonersen in Angelman Syndrome.
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2026-05-26
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2026-05-06
Long-term Extension of GTX-102 in Angelman Syndrome
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2026-05-06
A Natural History Study of Angelman Syndrome
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2026-02-18
IDMet (RaDiCo Cohort) (RaDiCo-IDMet)
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2026-02-12
Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2026-01-23
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2025-09-18
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2025-09-11
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2025-09-05
Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2025-08-22
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2025-08-03
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2025-07-18
Italian Angelman Syndrome Registry Protocol
Source: ClinicalTrials.gov: Angelman Syndrome | Last update posted in the last 14 days Published on 2025-06-26

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    Advice for parents new to an Angelman syndrome diagnosis







            
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            Published on 2025-04-08
        
    
    





            
        
                    
            
            

    Autism traits prevalent in children with Angelman; are stable with age







            
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            Published on 2025-04-02
        
    
    





            
        
                    
            
            

    Dealing with caregiver guilt is part of the Angelman journey







            
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            Published on 2025-03-11
        
    
    





            
        
                    
            
            

    Loss of consciousness can follow laughter in Angelman patients







            
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            Published on 2025-03-05
        
    
    





            
        
                    
            
            

    How we’ll be raising awareness on International Angelman Day







            
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            Published on 2025-02-11
        
    
    





            
        
                    
            
            

    Supporters gear up for Feb. 15 awareness day for Angelman







            
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            Published on 2025-02-05
        
    
    





            
        
                    
            
            

    Phase 3 Aspire trial of gene therapy GTX-102 doses 1st patient







            
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            Published on 2025-01-09
        
    
    





            
        
                    
            
            

    FAST’s Global Science Summit reminded us of the power of hope







            
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            Published on 2025-01-07
        
    
    





            
        
                    
            
            

    GTX-102 may help cognition in Angelman syndrome children, teens







            
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            Published on 2024-12-04
        
    
    





            
        
                    
            
            

    Burdine, Angelman researcher and parent, recognized for work







            
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            Published on 2024-11-06
        
    
    





            
        
                    
            
            

    Processing the complexities of our son’s happy demeanor







            
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            Published on 2024-11-05
        
    
    





            
        
                    
            
            

    Reflections after a year of early intervention therapy







            
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            Published on 2024-10-08
        
    
    





            
        
                    
            
            

    Children’s Colorado clinic named Angelman’s center of excellence







            
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            Published on 2024-10-02
        
    
    





            
        
                    
            
            

    Overcoming fear after an Angelman syndrome diagnosis







            
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            Published on 2024-09-10
        
    
    





            
        
                    
            
            

    Anxiety, repetitive behaviors predict sleep problems in Angelman







            
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            Published on 2024-09-04