Gaucher disease Treatment

There is currently no cure for Gaucher, but different therapies are available which may help treat many of the major symptoms.1

Enzyme Replacement Therapy (ERT)1

ERT is designed to work by replacing the missing or deficient enzyme. So the fatty molecules (cerebrosides) are removed by natural processes.

Substrate Reduction Therapy (SRT)1

SRT is designed to work by reducing the amount of fatty molecules (cerebrosides) in the body, so the enzyme has less work to do.

Patients with Gaucher disease may also be given medicines that help manage their symptoms (e.g. pain killers).2 These do not treat Gaucher disease itself but may help reduce the impact it has on the body.2

How is Gaucher disease inherited?

1. Nagral A. Gaucher disease. J Clin exp hepatol 2014;4(1):37-50.
2. Mignot C, et al. handb Clin Neurol. 2013;113:1709-15.
This leaflet hopes to answer questions about the inheritance of Gaucher disease.
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Watch a brief video about the causes of Gaucher disease.
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In this video Gaucher disease patients discuss how they were diagnosed.
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What are Lysosomal Storage Disorders?
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Visit the Spotlight on Gaucher YouTube channel to view Gaucher disease informational videos and stories from patients and families.
How is Gaucher disease diagnosed?
Symptoms Infographic
Symptoms Infographic
Download the Gaucher disease brochure for helpful information.

The information on this website is intended only to provide knowledge of Gaucher disease health topics. This information should not be used in place of advice from your GP or other healthcare professional. If in doubt please contact your doctor for advice. is intended for an International and US audience and has been produced by Shire. The website has been developed in accordance with industry and legal standards to provide information for the general public about Hunter Syndrome health topics. Shire makes every reasonable effort to include accurate and current information. However, the information provided on the website is not exhaustive.

Download the Gaucher disease toolkit for helpful information and questions to discuss with your doctor.
Symptoms Infographic