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What are the treatments for Gaucher disease?

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You can be born with Gaucher disease, which can lead to symptoms that include enlarged liver or spleen, bone problems, and tiredness. Mild cases may not need any treatment. Some treatments can only be used by adults. Others are designed for a specific type of Gaucher.

Most adults and kids with type 1, the most common form of Gaucher, get medication to replace or add enzymes that your liver or spleen can't make anymore. Adults also may take pills that cut down on fats that can build up in your liver, spleen, or bone marrow.

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

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