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Homozygous Familial Hypercholesterolemia (HoFH) is a rare, inherited disorder that causes extremely high levels of cholesterol in the blood. It is caused by a mutation in the LDL receptor gene, which is responsible for removing cholesterol from the blood. As a result, individuals with HoFH are at an increased risk of developing cardiovascular diseases such as heart attack and stroke. To treat HoFH, a variety of drugs are available, including statins, bile acid sequestrants, PCSK9 inhibitors, and cholesterol absorption inhibitors. Statins are the most commonly prescribed drugs for HoFH, as they reduce the production of cholesterol in the liver. Bile acid sequestrants bind to bile acids in the intestine, preventing them from being reabsorbed into the bloodstream. PCSK9 inhibitors block the action of the PCSK9 enzyme, which is responsible for breaking down LDL receptors. Finally, cholesterol absorption inhibitors reduce the absorption of cholesterol from the intestine.
Companies in the HoFH drug market include Amgen, Sanofi, Regeneron, Merck, and Pfizer. Show Less Read more