Normalization of low-density lipoprotein receptor expression in receptor defective homozygous familial hypercholesterolemia by inhibition of PCSK9 with alirocumab.

Proprotein convertase subtilisin kexin type-9 (PCSK9) inhibition using monoclonal antibodies (mAbs) lowers low-density lipoprotein cholesterol (LDL-C) by >50% in heterozygous familial hypercholesterolemia (HeFH) and non-FH dyslipidemic individuals [(1)][1]. Because PCSK9 is a circulating inhibitor