The goal of treatment is to reduce or prevent the buildup of GL-1. Gaucher disease type 1 can be effectively managed once a diagnosis has been made. Treatment options are available, including oral therapies.1,2

Why is treatment so important?

Gaucher disease is progressive and damage caused by Gaucher disease may be irreversible. Treatment can help alleviate certain symptoms and may prevent certain long term complications.3 Gaucher disease can be appropriately managed with treatment, including oral therapies.1,2 Talk to your doctor to find out which treatment option is right for you.

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There are two treatment approaches available for Gaucher Disease Type 1:

Substrate Reduction Therapy (SRT): Noninvasive Oral Capsule2,4
  • Oral therapy taken daily
  • Reduces the amount of GL-1 that is produced
Enzyme Replacement Therapy (ERT): Infusion2,5
  • Intravenous (IV) infusions usually given every 2 weeks at an infusion center, a doctor’s office, or at home as medically appropriate
  • Helps your body break down the excess GL-1
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Finding Gaucher disease assistance

You can use the National Gaucher Foundation’s treatment finder to find a Gaucher disease specialist.This link is provided as a resource only and does not constitute an endorsement by Sanofi Genzyme of the National Gaucher Foundation or its programming. Additional resources on this topic may be available and should be investigated. Sanofi Genzyme does not review or control the content of non-Sanofi Genzyme websites. Once you have a diagnosis of Gaucher disease, you and your doctor will determine the right treatment approach. The Sanofi Genzyme CareConnectPSS® team is here to help you learn more about Gaucher disease, treatments, and ongoing management. Visit CareConnectPSS.com, or call 1-800-745-4447 and Select Option 3.

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If you suspect Gaucher, get tested

Talk to your doctor -it’s a simple blood test

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Know the signs and symptoms

Identify Gaucher disease

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References:

1. Cox TM. Gaucher disease: clinical profile and therapeutic developments. Biol Targets Ther. 2010;4:299-313.

2. Pastores GM, Hughes DA. Gaucher disease. GeneReviews®. Seattle WA: NCBI Bookshelf. https://www.ncbi.nlm.nih.gov/books/NBK1269/?report=printable. Published July 27, 2000. Updated June 21, 2018. Accessed October 30, 2020.

3. Mistry PK, Capellini MD, Lukina E, et al. Consensus Conference: A reappraisal of Gaucher disease - diagnosis and disease management algorithms. Am J Hematol. 2011;86(1):110-115.

4. Shayman JA. Eliglustat tartrate: glucosylceramide synthase inhibitor treatment of type 1 Gaucher disease. Drugs Future. 2010;35(8):613-620.

5. Stirnemann J, Belmatoug N, Camou F, et al. A review of Gaucher disease pathophysiology, clinical presentation and treatments. Int J Mol Sci. 2017;18(2)