Abstract
Background and Aims: Homozygous familial hypercholesterolaemia (HoFH) is a genetic disorder characterised by severely elevated plasma LDL cholesterol (LDL-C) levels and development of premature atherosclerotic cardiovascular disease. Robust and durable LDL-C lowering is essential, but additional reductions beyond what can be achieved with statins and ezetimibe are required. We evaluated the LDL-C–lowering effect and safety of inclisiran in an open-label, single-arm, multicentre pilot study in HoFH patients receiving high-intensity statin plus ezetimibe.
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