Existing and emerging therapies for the treatment of familial hypercholesterolemia

Familial hypercholesterolemia (FH), an autosomal dominant disorder of LDL metabolism that is characterized by elevated LDL cholesterol, is commonly encountered in patients with atherosclerotic coronary heart disease. Combinations of cholesterol lowering therapies are often used to lower LDL cholesterol in patients with familial hypercholesterolemia; however, current treatment goals for LDL cholesterol are rarely achieved in patients with homozygous FH, and are difficult to achieve in patients with heterozygous FH. Therapies that lower LDL cholesterol through LDL receptor-mediated mechanisms have thus far been largely ineffective in patients with homozygous FH, particularly in those with negligible (