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Two New Drugs for Homozygous Familial Hypercholesterolemia:  Managing Benefits and Risks in a Rare Disorder

Robert J. Smith, MD1,2; William R. Hiatt, MD3,4
[+] Author Affiliations
1Department of Medicine, Alpert Medical School of Brown University, Providence, Rhode Island
2Ocean State Research Institute, Providence Veterans Administration Medical Center, Providence, Rhode Island
3Division of Cardiology, The University of Colorado School of Medicine, Aurora
4CPC Clinical Research, Aurora, Colorado
JAMA Intern Med. 2013;173(16):1491-1492. doi:10.1001/jamainternmed.2013.6624.
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Homozygous familial hypercholesterolemia (HoFH) has the highest cardiovascular risk of any known atherosclerotic disease, with severe and progressive cardiovascular events frequently presenting in childhood.1 Affected individuals have either the same mutation in both low-density lipoprotein (LDL) receptor alleles (true homozygotes) or distinct loss-of-function mutations in each LDL receptor allele (technically compound heterozygotes). The pathophysiologic mechanism is strongly related to increased LDL cholesterol (LDL-C) levels and LDL receptor status.

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