The Medical Letter on Drugs and Therapeutics
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Two New Drugs for Homozygous Familial Hypercholesterolemia

The FDA has approved mipomersen (Kynamro – Genzyme) and lomitapide (Juxtapid – Aegerion), each in addition to a low-fat diet and other lipid-lowering medications, to reduce cholesterol levels in patients with homozygous familial hypercholesterolemia (HoFH).

THE DISORDER — Familial hypercholesterolemia is an inherited condition most commonly caused by defects in the low-density lipoprotein (LDL) receptor gene that result in very high levels of LDL cholesterol.1 Homozygous familial hypercholesterolemia (HoFH) is the most severe form of the disease; without treatment, cardiovascular disease and death can occur in childhood. The prevalence of HoFH has been estimated to be about one per million persons; it affects about 300 patients in the US.

STANDARD TREATMENT — The standard treatment for patients with HoFH includes a low-fat diet, high doses of ... more      

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