Heterozygous familial hypercholesterolemia is a common genetic disorder that leads to premature coronary artery disease. Despite aggressive lipid-lowering therapy, many patients with heterozygous familial hypercholesterolemia fail to achieve optimal low-density lipoprotein cholesterol (LDL-C) goals. We evaluated mipomersen, an apolipoprotein B synthesis inhibitor, to further lower LDL-C in pati...

The hyperlipidemia observed in familial hypercholesterolemia can be reduced by portacaval anastomosis. We report the effects of a portacaval shunt on hepatic morphology and biosynthetic pathways crucial to hepatic cholesterol homeostasis in homozygous receptor-negative familial hypercholesterolemia. Portacaval anastomosis was associated with a dramatic change in hepatocyte morphology, 28% reduc...