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
Related Articles
- EP ID EP217508
- DOI -
- Views 85
- Downloads 0
A STUDY ON ASSOCIATION BETWEEN CIGARETTE SMOKING AND GASTRIC CANCER
Objective: The study was aimed at assessing the association of smoking with gastric cancer since gastric cancer remains to be one of the leading causes of death from cancer worldwide in spite of advanced treatment modali...
A STUDY ON NUTRITIONAL STATUS OF SCHOOL CHILDREN IN RURAL, SEMI URBAN AND URBAN AREAS OF TAMIL NADU.
BACKGROUND: The health and nutritional status of children is an index of national investment in the development of its future manpower. Malnutrition affects the child’s physical and cognitive growth and increases the sus...
Malignant parotid tumor with sialadenitis - a diagnostic dilemma
Malignant parotid tumor associated with chronic sialadenitis is rare entity. These tumors mostly arise from epithelial component of chronic sialadenitis. Chronic sialadenitis of parotid with malignant epithelial tumor ev...
Intrapleural instillation of Tranexamic acid for the control of postoperative bleeding following thoracic surgical procedures for chronic inflammatory pleural/pulmonary diseases - A Randomised prospective clinical study
Context Postoperative bleeding following open thoracic surgical procedures for chronic inflammatory pleural/pulmonary diseases (CIPPD) is adding considerable morbidity and mortality by increasing the need for more blood...
A STUDY TO DETERMINE THE ASSOCIATION BETWEEN THE KELLGREN-LAWRENCE GRADE AND WOMAC SCORE IN POPULATION WITH OSTEOARTHRITIC KNEE
Objective:To determine the association between Kellgren-Lawrence radiological grade and WOMAC scores among population with osteoarthritic knee. Study design: This is a cross sectional quantitative prospective study desig...