Miglustat is a drug used to treat endocrine and metabolic disorders. It is used to treat Gaucher disease type 1, a rare genetic disorder that affects the body's ability to break down and store fat. It is also used to treat Niemann-Pick disease type C, a rare genetic disorder that affects the body's ability to break down and store cholesterol. Miglustat works by inhibiting the enzyme glucosylceramide synthase, which is responsible for the production of glucosylceramide, a molecule that accumulates in the cells of patients with these diseases. Miglustat is available in both oral and intravenous formulations. It is marketed by several pharmaceutical companies, including Actelion Pharmaceuticals, Sanofi, and Shire. It is also available as a generic drug in some countries. Companies in the Miglustat market include Actelion Pharmaceuticals, Sanofi, Shire, and generic drug manufacturers. Show Less Read more
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