Abstract
Background and Aims: Background: Homozygous familial hypercholesterolemia (HoFH) is characterized by severely elevated low-density lipoprotein-cholesterol (LDL-C) associated with extreme cardiovascular risk. Lomitapide is a lipid-lowering agent indicated as adjunct therapy for HoFH patients. This study aimed at evaluating the real-world long-term efficacy and safety of lomitapide in the management of HoFH.
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