Stephen J. Schueler, M.D.

Overview Incidence Symptoms Evaluation Treatment drugs questions for doctor Home Care taking control warning signs Outlook Underlying Cause Types Genetics

Gaucher's Disease Treatment

There is no cure for Gaucher's disease. Treatment for Gaucher's disease includes enzyme replacement therapy, medications to control symptoms, and surgical removal of the spleen.

Treatment for Gaucher's disease includes:

  • Enzyme replacement therapy for Gaucher's disease:
  • Additional medications for Gaucher's disease:
  • Splenectomy for Gaucher's disease:
    • Removal of the spleen
  • Bone marrow transplantation

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.

Gaucher's Disease Questions For Doctor

The following are some important questions to ask before and after the treatment of Gaucher's disease.

Questions to ask before treatment:

  • What are my treatment options?
  • What are the risks associated with treatment?
  • Do I need to stay in the hospital?
    • How long will I be in the hospital?
  • What are the complications I should watch for?
  • How long will I be on medication?
  • What are the potential side effects of my medication?
  • Does my medication interact with nonprescription medicines or supplements?
  • Should I take my medication with food?

Questions to ask after treatment:
  • How should I change my diet?
  • When can I resume my normal activities?
  • When can I return to work?
  • How often will I need to see my doctor for checkups?
  • Will my children have this condition?
  • What local support and other resources are available?

Continue to Gaucher's Disease Home Care

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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