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Miglustat


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This WisdomCard managed by: Chris Miller

Miglustat is a medication utilized to treat Gaucher disease in patients who cannot be treated with enzyme replacement therapy using imiglucerase.1 Type 1 Gaucher is inherited from both parents, and people affected by the disease are missing an enzyme that breaks down a chemical called glucosylceramide.2

Fast Facts:

  • -- Sold as the generic for the brand Zavesca.
  • -- Works by preventing the body from making glucosylceramide.2
  • -- Side effects include bleeding gums, blood in the urine or stool, and numbness or tingling.2
  • -- Miglustat has been tested for the treatment of Niemann Pick disease.

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GuideWisdom and Fast Facts References

  1. Wikipedia: Miglustat
  2. 2.0 2.1 2.2 Micromedex (via Mayo Clinic): Miglustat (Oral Route)

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