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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Phase 1/2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS)FOXG1 disorderEnrolling by invitationPhase 1 / Phase 22026-07-24NCT07293546
Primary Sclerosing Cholangitis in ChildrenCholangitis, Sclerosing, Digestive System Diseases, bile duct disorder, biliary tract disorder, cholangitis, liver disorder, primary sclerosing cholangitisRecruitingNot stated2026-07-14NCT04181138
Biliary Atresia Study in Infants and ChildrenCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Abnormalities, Digestive System Diseases, bile duct disorder, biliary atresia, biliary tract disorder, cholestasisRecruitingNot stated2026-06-04NCT00345553
Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiencycongenital disorder of deglycosylation 1Active, not recruitingPhase 32026-05-04NCT06199531