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Reviews in Cardiovascular Medicine  2016, Vol. 17 Issue (1-2): 16-27     DOI: 10.3909/ricm0854
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Emerging Treatments for Heterozygous and Homozygous Familial Hypercholesterolemia
Seth J. Baum1, Daniel Soffer2, P. Barton Duell3
1 Preventive Cardiology, Inc., Boca Raton, FL
2 University of Pennsylvania Health System, Philadelphia, PA
3 Oregon Health and Science University, Portland, OR
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Abstract:
Familial hypercholesterolemia (FH) is an autosomal co-dominant disorder marked by extremely high low-density lipoprotein (LDL) cholesterol levels and concomitant premature vascular disease. FH is caused by mutations that most commonly affect three genes integrally involved in the LDL receptor’s ability to clear LDL particles from the circulation. Primary intervention efforts to lower LDL cholesterol have centered on therapies that upregulate the LDL receptor. Unfortunately, most patients are insufficiently responsive to traditional LDL-lowering medications. This article focuses primarily on the clinical management of homozygous FH.
Key words:  Familial hypercholesterolemia      Low-density lipoprotein cholesterol      Lipid-lowering therapy      Mipomersen      Lomitapide      LDL apheresis      PCSK9 inhibitors     
Published:  30 March 2016     

Cite this article: 

Seth J. Baum, Daniel Soffe, P. Barton Duell. Emerging Treatments for Heterozygous and Homozygous Familial Hypercholesterolemia. Reviews in Cardiovascular Medicine, 2016, 17(1-2): 16-27.

URL: 

https://rcm.imrpress.com/EN/10.3909/ricm0854     OR     https://rcm.imrpress.com/EN/Y2016/V17/I1-2/16

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