Results for tag: "Niemann Pick Disease Drug"

Niemann Pick Disease (NPD) is a rare, inherited metabolic disorder that affects the body's ability to metabolize lipids. It is classified as an endocrine and metabolic disorder, and is treated with drugs that target the underlying metabolic defect. These drugs are designed to reduce the accumulation of lipids in the body, and to improve the patient's quality of life. Commonly used drugs for NPD include miglustat, cyclodextrin, and enzyme replacement therapy. Miglustat is a small molecule drug that works by inhibiting the enzyme glucosylceramide synthase, which is responsible for the accumulation of lipids in the body. Cyclodextrin is a complex carbohydrate that binds to lipids, allowing them to be excreted from the body. Enzyme replacement therapy is a form of gene therapy that replaces the defective enzyme responsible for the accumulation of lipids. The Niemann Pick Disease Drug market is a specialized market, with a limited number of companies offering treatments. These include Sanofi, Actelion Pharmaceuticals, and Shire Pharmaceuticals. Show Less Read more