Organization Overview
Alternative names
givosiran (Givlaari) (2019 NDA)
lumasiran (Oxlumo) (2020 NDA)
patisiran (ONPATTRO) (2018 NDA)
vutrisiran (Amvuttra) (2022 NDA)
alpha 1-Antitrypsin Deficiency (Phase 2)
Amyloid Neuropathies (Phase 3)
Amyloid Neuropathies, Familial (Phase 3)
Amyloidosis (Phase 3)
Amyloidosis, Familial (Phase 3)
Atypical Hemolytic Uremic Syndrome (Phase 2)
Azotemia (Phase 2)
Cardiomyopathies (Phase 3)
Communicable Diseases (Phase 2)
Coproporphyria, Hereditary (Phase 3)
Disease Progression (Phase 3)
Fatty Liver (Phase 1)
Glomerulonephritis (Phase 2)
Glomerulonephritis, IGA (Phase 2)
Hemoglobinuria (Phase 2)
Hemoglobinuria, Paroxysmal (Phase 2)
Hemolysis (Phase 2)
Hemolytic-Uremic Syndrome (Phase 2)
Hemophilia A (Phase 2)
Hemophilia B (Phase 2)
Hepatitis (Phase 2)
Hepatitis A (Phase 2)
Hepatitis B (Phase 2)
Hepatitis B, Chronic (Phase 2)
Hepatitis, Chronic (Phase 2)
Hypercholesterolemia (Phase 1)
Hyperoxaluria, Primary (Phase 3)
Hypertension (Phase 1)
Infections (Phase 2)
Kidney Diseases (Phase 2)
Liver Diseases (Phase 2)
Neoplasms (Phase 1)
Non-alcoholic Fatty Liver Disease (Phase 1)
Polyneuropathies (Phase 3)
Porphyria, Acute Intermittent (Phase 3)
Porphyria, Erythropoietic (Phase 3)
Porphyrias (Phase 3)
Porphyrias, Hepatic (Phase 3)
Porphyria, Variegate (Phase 3)
Respiratory Syncytial Virus Infections (Phase 2)
Syndrome (Phase 2)
Virus Diseases (Phase 2)