Angelman Syndrome
Pipeline by Development Stage
Competitive Landscape
6 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (15)
Total enrollment: 939 patients across 15 trials
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
Long-term Extension of GTX-102 in Angelman Syndrome
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
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.
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants
Angelman Syndrome (AS) Biomarker Study
A Natural History Study of Angelman Syndrome
Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi Syndrome
Angelman Syndrome Natural History Study-FAST UK
Angelman Syndrome Video Assessment (ASVA) Source Material Study