GTX-102 ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
201Angelman syndrome1

201. Angelman syndrome


Clinical trials : 24 Drugs : 35 - (DrugBank : 9) / Drug target genes : 22 - Drug target pathways : 20
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agemin
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PhaseCountries
1NCT04259281
(ClinicalTrials.gov)
February 24, 202031/1/2020A Study of the Safety and Tolerability of GTX-102 in Children With Angelman SyndromeA Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)Angelman SyndromeDrug: GTX-102GeneTX Biotherapeutics, LLCUltragenyx Pharmaceutical IncRecruiting4 Years17 YearsAll20Phase 1/Phase 2United States;Canada;United Kingdom