MIPOMERSEN – A NOVEL ANTISENSE DRUG FOR FAMILIAL HYPERCHOLESTEROLEMIA
Journal Title: Stanley Medical Journal - Year 2015, Vol 2, Issue 2
Abstract
Primary mature cystic teratomas are Familial Hypercholesterolemia (FH) is an autosomal dominant disorder characterized by elevated plasma levels of low density cholesterol (LDL-C), is caused by mutations in the LDL receptor gene. Total cholesterol levels are usually >500 mg/dL and can be as high as 1000 mg/dL. Most patients with homozygous FH present in childhood with cutaneous xanthomas on the hands, wrists, elbows, knees, heels, or buttocks. The devastating complication of homozygous FH is accelerated atherosclerosis, which can result in disability and death in childhood1. Treatment is often complex and complicated. FH patients requires Statins (HMG co-reductase inhibitors), in combination with the addition of a cholesterol absorption inhibitor and/or bile acid sequestrant. Nicotinic acid is also added sometimes. These drugs are given at higher doses and hence adverse effects by these drugs such as nausea, myalgia, liver damage, pigmentation (by niacin) are common 2. Treatment is largely unsatisfactory, none of the currently available hypolidemic drug alters natural history of the disease process considerably. There is an unmet need of drug development for this condition, and hope arrived with the discovery of Mipomersen, an antisense oligonucleotide molecule approved recently by FDA for familial hypercholesterolemia.
Authors and Affiliations
Krishnan V, Vasanthira K
Keywords
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