Novel therapies for treating familial hypercholesterolemia.

Familial hypercholesterolemia is an inherited disorder associated with early accelerated atherosclerosis with morbidity and mortality resulting from premature cardiovascular disease. Affected individuals have extreme elevations in low-density lipoprotein cholesterol levels. Patients usually do not achieve target reductions in cholesterol levels with conventional antihyperlipidemic pharmacotherapy. This unmet need has resulted in the recent development and approval of novel therapies targeting different cholesterol pathways. This article briefly summarizes familial hypercholesterolemia and then discusses the newer pharmacotherapies available in the management of familial hypercholesterolemia.