Results for tag: "Fabry Disease Drug"

Fabry Disease is a rare, inherited disorder caused by the deficiency of an enzyme called alpha-galactosidase A. This deficiency leads to the accumulation of a fatty substance called globotriaosylceramide (GL-3) in the body's cells, resulting in a wide range of symptoms. Endocrine and Metabolic Disorders Drugs are used to treat Fabry Disease, including enzyme replacement therapy (ERT) and chaperone therapy. ERT is the most common treatment, and involves the administration of a recombinant form of the missing enzyme. Chaperone therapy is a newer treatment option, and involves the use of small molecules to help the body's cells process GL-3. The Fabry Disease Drug market is a rapidly growing segment of the Endocrine and Metabolic Disorders Drugs market. Companies in the market include Sanofi, Amicus Therapeutics, Shire, and Pfizer. Show Less Read more