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Can enzyme replacement therapy treat Gaucher's disease?

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Enzyme replacement therapy is a treatment option for people who have type 1 Gaucher's disease and some with type 3. It helps reduce anemia and shrinks an enlarged spleen or liver.

Enzyme replacement therapy medications may include:

  • Imiglucerase (Cerezyme)
  • Taliglucerase alfa (Elelyso)
  • Velaglucerase alfa (VPRIV)

From: Gaucher Disease WebMD Medical Reference

SOURCES:

National Institute of Neurological Disorders and Stroke: "What is Gaucher Disease?"

Genetics Home Reference: "Gaucher disease."

National Gaucher Foundation: "About Gaucher Disease."

Kliegman, R.M.   19th edition, Saunders Elsevier, 2011. Nelson Textbook of Pediatrics,

National Human Genome Research Institute: "Learning About Gaucher Disease."

Orphanet: "Gaucher disease - ophthalmoplegia - cardiovascular calcification."

Weinreb, N.J.  , December 2008. American Journal of Hematology

Gauchers Association: "Living with Gaucher Disease."

Reviewed by Sabrina Felson on November 07, 2018

SOURCES:

National Institute of Neurological Disorders and Stroke: "What is Gaucher Disease?"

Genetics Home Reference: "Gaucher disease."

National Gaucher Foundation: "About Gaucher Disease."

Kliegman, R.M.   19th edition, Saunders Elsevier, 2011. Nelson Textbook of Pediatrics,

National Human Genome Research Institute: "Learning About Gaucher Disease."

Orphanet: "Gaucher disease - ophthalmoplegia - cardiovascular calcification."

Weinreb, N.J.  , December 2008. American Journal of Hematology

Gauchers Association: "Living with Gaucher Disease."

Reviewed by Sabrina Felson on November 07, 2018

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