Migalastat

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Migalastat

Migalastat (pronounced as mi-ga-las-tat) is a medication used for the treatment of Fabry disease, a rare genetic disorder. It is marketed under the brand name Galafold.

Etymology

The name "Migalastat" is derived from its chemical structure, which is a derivative of 1-deoxygalactonojirimycin. The "Miga-" prefix is a contraction of "mimetic galactose", referring to the drug's mechanism of action as a pharmacological chaperone that mimics the structure of the sugar galactose.

Usage

Migalastat is used to treat adults with Fabry disease who have a specific genetic mutation that is responsive to this medication. It works by helping the body produce an enzyme called alpha-galactosidase A, which is deficient in people with Fabry disease.

Related Terms

  • Fabry disease: A rare genetic disorder that results from the buildup of a particular type of fat, called globotriaosylceramide, in the body's cells.
  • Pharmacological chaperone: A small molecule that binds to and stabilizes a protein, helping it to fold and function correctly.
  • Alpha-galactosidase A: An enzyme that breaks down certain fats in the body. Deficiency of this enzyme leads to Fabry disease.

See Also

  • Enzyme replacement therapy: A medical treatment for some diseases caused by the deficiency or absence of a specific enzyme in the body.
  • Genetic disorder: A disease caused by abnormalities in an individual's genetic material.

External links

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