Existing and emerging therapies for the treatment of familial hypercholesterolemia

Familial hypercholesterolemia (FH), an autosomal dominant disorder of LDL metabolism that is characterized by elevated LDL-cholesterol, is commonly encountered in patients with atherosclerotic coronary heart disease. Combinations of cholesterol-lowering therapies are often used to lower LDL-choleste...

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Bibliographic Details
Published in:Journal of lipid research 2021-01, Vol.62, p.100060, Article 100060
Main Author: Rosenson, Robert S.
Format: Article
Language:English
Subjects:
angiopoietin-like protein 3
Anticholesteremic Agents - therapeutic use
atherosclerotic cardiovascular disease
Cholesterol, LDL - blood
Cholesterol, LDL - metabolism
cholesterol-lowering therapies
familial hypercholesterolemia
genetics
Humans
Hydroxymethylglutaryl-CoA Reductase Inhibitors - therapeutic use
Hyperlipoproteinemia Type II - drug therapy
LDL
LDL receptor
lipoprotein (a)
proprotein convertase subtilisin Kexin 9
Receptors, LDL - genetics
Receptors, LDL - metabolism
Thematic Review Series
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Summary:Familial hypercholesterolemia (FH), an autosomal dominant disorder of LDL metabolism that is characterized by elevated LDL-cholesterol, is commonly encountered in patients with atherosclerotic coronary heart disease. Combinations of cholesterol-lowering therapies are often used to lower LDL-cholesterol in patients with FH; however, current treatment goals for LDL-cholesterol are rarely achieved in patients with homozygous FH (HoFH) and are difficult to achieve in patients with heterozygous FH (HeFH). Therapies that lower LDL-cholesterol through LDL receptor-mediated mechanisms have thus far been largely ineffective in patients with HoFH, particularly in those with negligible (
ISSN:0022-2275
1539-7262
1539-7262
DOI:10.1016/j.jlr.2021.100060