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LJPC-401 is a synthetic peptide formulation of human hepcidin developed for the treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, and sickle cell disease. Hepcidin is a naturally occurring peptide hormone that regulates iron metabolism by suppressing iron release and absorption in the body. By mimicking endogenous hepcidin activity, LJPC-401 reduces serum iron levels and prevents excessive accumulation of iron in organs like the liver and heart. Clinical studies have shown that LJPC-401 significantly decreases transferrin saturation and reduces the need for phlebotomy procedures in patients with hereditary hemochromatosis. The drug has also been investigated for use in beta thalassemia (including transfusion-dependent cases) and sickle cell disease[4][5][6][7][8].
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