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Rader - Figure 20 - Current therapies for lowering LDL converge on upregulation of the hepatic LDL receptor

Therapy for patients with heterozygous FH in general is simply the standard lipid-lowering therapy currently available, meaning high-dose statins, and then, if needed, cholesterol absorption inhibitors and bile acid sequestrants, all of which target the normal LDL receptor allele, resulting in up-regulation of the LDL receptor and lowering of circulating LDL-C.  This is how lipid-lowering therapy should work, and of course many heterozygous FH patients are reasonably responsive to standard LDL-lowering therapy, based on up-regulation of the LDL receptor.
Rader J Clin Lipidol. 2012;
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