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Hope for Inherited, Dangerously High Cholesterol

New drug reduces LDL in people with rare genetic condition, researchers say

SUNDAY, March 14 (HealthDay News) -- A new drug called mipomersen reduced low-density lipoprotein (LDL) "bad" cholesterol by nearly 25 percent when added to current therapy in people with a rare genetic condition that causes extremely high cholesterol, a new study finds.

Mipomersen is designed to decrease the formation of apolipoprotein B (the main structural protein in LDL cholesterol) and its release from the liver or intestine. This reduces circulating LDL cholesterol concentrations.

Inherited high cholesterol (homozygous familial hypercholesterolemia) affects one in every one million people. Patients with the disorder have severely elevated LDL cholesterol and a high risk of early cardiovascular disease. If untreated, these patients rarely live past the age of 30.

This phase 3 clinical trial included 51 patients with homozygous FH who were already taking lipid-lowering drugs, including high-dose statins. The patients were randomly selected to receive either 200 milligrams of mipomersen or placebo per week for 26 weeks.

At the end of the treatment period, LDL cholesterol levels had decreased 24.7 percent in the mipomersen group, compared with 3.3 percent in the placebo group.

"Mipomersen could be a valuable addition to the drugs used in the management of homozygous FH and should prove useful in the management of other forms of severe refractory hypercholesterolemia," wrote an international team of researchers led by Frederick Raal of the University of Witwatersrand, Johannesburg, South Africa.

The study was published online March 13 in advance of print publication this week in The Lancet, and was slated to be presented Saturday at the annual meeting of the American College of Cardiology in Atlanta.

More information

The U.S. National Library of Medicine has more about homozygous familial hypercholesterolemia.

-- Robert Preidt

SOURCE: The Lancet, news release, March 13, 2010

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