Gaucher disease treatment
There is currently no cure for Gaucher disease, but different therapies are available which may help treat many of the major symptoms.1
The treatments described below have some limitations and are not suitable for everyone; for further information, please contact your doctor.
Enzyme replacement therapy (ERT)2,3
ERT was first approved for treatment of Gaucher disease in 1991.4 It is designed to
work by replacing the deficient enzyme, so the fatty substance glucocerebroside is removed by natural processes. ERT can be used to treat children and adults with Gaucher disease.2,3
Substrate reduction therapy (SRT)5,6
SRT was first approved for treatment of Gaucher disease in 2002.5 It is designed to work by reducing the amount of glucocerebroside in the body, so the enzyme has less work to do. SRT can be used to treat adults with Gaucher disease.5,6
Patients with Gaucher disease may also be given medicines that help manage their symptoms (e.g. pain killers).1 These do not treat Gaucher disease itself but may help reduce the impact it has on the body.1
- Pastores GM, Hughes DA. Gaucher Disease. In: Adam MP, et al., editors. GeneReviews® [Internet]. Seattle, Washington: University of Washington, Seattle; 1993–2018. Available at: https://www.ncbi.nlm.nih.gov/books/NBK1269/; Accessed: August 2019.
- Velaglucerase alfa Summary of Product Characteristics.
- Imiglucerase Summary of Product Characteristics.
- US Food and Drug Administration (FDA). FDA Approved Drug Products: alglucerase. Available at: https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=020057; Accessed August 2019.
- Miglustat Summary of Product Characteristics.
- Eliglustat Summary of Product Characteristics.
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.
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