Abstract
Several new or emerging drugs for dyslipidemia owe their existence, in part, to human genetic evidence, such as observations in families with rare genetic disorders or in Mendelian randomization studies. Much effort has been directed to agents that reduce LDL (low-density lipoprotein) cholesterol, triglyceride, and Lp[a] (lipoprotein[a]), with some sustained programs on agents to raise HDL (high-density lipoprotein) cholesterol. Lomitapide, mipomersen, AAV8.TBG.hLDLR, inclisiran, bempedoic acid, and gemcabene primarily target LDL cholesterol. Alipogene tiparvovec, pradigastat, and volanesorsen primarily target elevated triglycerides, whereas evinacumab and IONIS-ANGPTL3-LRx target both LDL cholesterol and triglyceride. IONIS-APO(a)-LRx targets Lp(a).
Keywords:
atherosclerosis; dyslipidemias; humans; lipoprotein(a); oligonucleotides, antisense.
Publication types
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Research Support, N.I.H., Extramural
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Research Support, Non-U.S. Gov't
MeSH terms
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Antibodies, Monoclonal / therapeutic use
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Anticholesteremic Agents / therapeutic use
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Benzimidazoles / therapeutic use
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Caproates / therapeutic use
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Cholesterol, HDL / blood
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Cholesterol, LDL / blood
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Diacylglycerol O-Acyltransferase / antagonists & inhibitors
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Dicarboxylic Acids / therapeutic use
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Dyslipidemias / blood
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Dyslipidemias / drug therapy*
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Ezetimibe / therapeutic use
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Fatty Acids / therapeutic use
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Genetic Therapy
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Humans
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Hypertriglyceridemia / drug therapy
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Hypolipidemic Agents / therapeutic use*
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Lipoprotein(a) / blood
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Oligonucleotides / therapeutic use
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RNA, Small Interfering / therapeutic use
Substances
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ALN-PCS
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Antibodies, Monoclonal
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Anticholesteremic Agents
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BMS201038
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Benzimidazoles
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Caproates
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Cholesterol, HDL
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Cholesterol, LDL
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Dicarboxylic Acids
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Fatty Acids
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Hypolipidemic Agents
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ISIS 304801
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Lipoprotein(a)
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Oligonucleotides
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RNA, Small Interfering
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8-hydroxy-2,2,14,14-tetramethylpentadecanedioic acid
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mipomersen
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gemcabene
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Diacylglycerol O-Acyltransferase
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Ezetimibe
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evinacumab