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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseActive, not recruitingPhase 2 / Phase 32026-06-23NCT05031780
Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingPhase 32026-03-05NCT06165341