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How does enzyme replacement therapy (ERT) help patients with Gaucher disease?

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Gaucher disease is an inherited condition that may lead to enlarged liver or spleen, among other symptoms. ERT can often help:

There are three ERT drugs, which you get through an IV:

  • Shrink the size of your liver or spleen if they get too large
  • Ease anemia that makes you weak or tired
  • Build bone strength to ease pain and prevent breaks
  • Imiglucerase (Cerezyme)
  • Taliglucerase alfa (Elelyso)
  • Velaglucerase alfa (VPRIV)

From: Treatments for Gaucher Disease WebMD Medical Reference

SOURCES:

Mayo Clinic: "Gaucher Disease: Treatments and drugs."

National Gaucher Foundation.

National Institute of Neurological Disorders and Strokes.

Gaucher Association: "What is Type 3 Gaucher Disease?"

Genetic Disease Foundation.

Eghbali-Fatourechi, G. , published online December 2014. Journal of Diabetes and Metabolic Disorders

National Gaucher Foundation of Canada: "Treatment Options."

Cochrane Review 2017.

Reviewed by Louise Chang on August 05, 2018

SOURCES:

Mayo Clinic: "Gaucher Disease: Treatments and drugs."

National Gaucher Foundation.

National Institute of Neurological Disorders and Strokes.

Gaucher Association: "What is Type 3 Gaucher Disease?"

Genetic Disease Foundation.

Eghbali-Fatourechi, G. , published online December 2014. Journal of Diabetes and Metabolic Disorders

National Gaucher Foundation of Canada: "Treatment Options."

Cochrane Review 2017.

Reviewed by Louise Chang on August 05, 2018

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What medicines can you take for Gaucher disease?

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