title: The promise of proprotein convertase subtilisin/kexin 9 inhibitors for the treatment of familial hypercholesterolemia.
abstract: Familial hypercholesterolemia comprises a constellation of genetic disorders resulting in very high cholesterol levels since childhood. If untreated, it is associated with accelerated atherosclerosis and premature cardiovascular disease. It has been shown that if aggressive cholesterol lowering is achieved in familial hypercholesterolemia, the incidence of cardiovascular disease can be lowered. However, currently approved pharmacological therapies are not able to lower cholesterol to optimal levels in a large number of these patients. Proprotein convertase subtilisin/kexin 9 inhibitors are a new class of cholesterol-lowering medications that can significantly reduce cholesterol levels in these patients especially those with at least some functioning low-density lipoprotein receptors. In this article, we will briefly review familial hypercholesterolemia and the role of proprotein convertase subtilisin/kexin 9 inhibitors in this condition.
authors.affiliation: Sections of Cardiovascular Research, Department of Medicine, Baylor College of Medicine, 6565 Fannin St. Suite B157, Houston, TX, 77030, USA, Pokharel@bcm.edu.
authors.forename: Yashashwi
authors.initials: Y
authors.lastname: Pokharel
authors.forename: Salim S
authors.initials: SS
authors.lastname: Virani
authors.forename: Christie M
authors.initials: CM
authors.lastname: Ballantyne
mesh_terms: D017209:Apoptosis; D004791:Enzyme Inhibitors / Q000627:therapeutic use*; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000188:drug therapy* / Q000201:enzymology; D000071449:Proprotein Convertase 9; D043484:Proprotein Convertases / Q000037:antagonists & inhibitors* / Q000378:metabolism; D012697:Serine Endopeptidases / Q000378:metabolism
chemical_list: D004791:Enzyme Inhibitors; C472125:PCSK9 protein, human; D000071449:Proprotein Convertase 9; D043484:Proprotein Convertases; D012697:Serine Endopeptidases