New Therapeutic Approaches for Familial Hypercholesterolemia.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 29414257.
- Also identified by DOI 10.1146/annurev-med-051215-030943.
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Abstract
Familial hypercholesterolemia (FH) is a common genetic condition characterized by elevated plasma levels of low-density lipoprotein cholesterol (LDL-C), premature atherosclerotic cardiovascular disease, and considerable unmet medical need with conventional LDL-C-lowering therapies. Between 2012 and 2015, the US Food and Drug Administration approved four novel LDL-C-lowering agents for use in patients with FH based on the pronounced LDL-C-lowering efficacy of these medicines. We review the four novel approved agents, as well as promising LDL-C-lowering agents in clinical development, with a focus on their mechanism of action, efficacy in FH cohorts, and safety.
Medical subject headings
- Anticholesteremic Agents
- Hyperlipoproteinemia Type II