Stephen J. Schueler, M.D.

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Gaucher's Disease Drugs

Medications used to treat Gaucher's disease include:

  • Imiglucerase:
    • A synthetic replacement for the enzyme that breaks down toxic molecules
  • Miglustat:
    • Reduces the production of toxic molecule
  • prGCD
    • New drug for Gaucher's pending approval by FDA
    • Glucocerebrosidase (GCD) is used as enzyme replacement therapy for patients with Gaucher's.

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Last Updated: Dec 14, 2010 References
Authors: Stephen J. Schueler, MD; John H. Beckett, MD; D. Scott Gettings, MD
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PubMed Gaucher's Disease References
  1. Brady RO. Gaucher's disease: past, present and future. Baillieres Clin Haematol. 1997 Dec;10(4):621-34. [9497855]
  2. Germain DP. Gaucher's disease: a paradigm for interventional genetics. Clin Genet. 2004 Feb;65(2):77-86. [14984463]
  3. Niederau C, Haussinger D. Gaucher's disease: a review for the internist and hepatologist. Hepatogastroenterology. 2000 Jul-Aug;47(34):984-97. [11020862]
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