Treating Gaucher disease type 1

Gaucher disease type 1 can be effectively managed once a diagnosis has been made. The goal of treatment is to reduce or prevent the buildup of GL-1.

There are two treatment approaches available to help manage some of the signs and symptoms of Gaucher disease type 1: substrate reduction therapy (SRT)—an oral option, and enzyme replacement therapy (ERT)—an infusion.

Oral therapy Substrate Reduction Therapy (SRT); Noninvasive oral capsule
  • Oral therapy taken daily
  • Reduces the amount of GL-1 that is produced
Learn more about an SRT oral therapy »
Intravenous (IV) infusions Enzyme Replacement Therapy (ERT): Infusion
  • 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
Learn more about an ERT treatment option »

Remember the goal of treatment: Reduce or prevent the buildup of GL-1.

Why is treatment so important?

Gaucher disease type 1 is a rare and progressive disease, which means it can worsen over time. Treatment can help alleviate certain symptoms and may help prevent certain long term complications.

Gaucher disease type 1 is manageable. Gaucher disease can be appropriately managed with treatment, including oral therapy. Talk to your doctor to find out which treatment option is right for you.

Finding Gaucher disease assistance

You can use the National Gaucher Foundation’s treatment finder to find a Gaucher disease specialist.

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, treatment, and ongoing management. Visit, or call 1-800-745-4447 and Select Option 3, M-F, 8:00 AM to 6:00 PM EST.

Melina, Argentina, Gaucher Disease Type 1 Patient

Support is available

Our CareConnectPSS team is here for patients and their families affected by Gaucher disease.

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It’s easy to get tested for Gaucher disease

Early diagnosis and timely treatment of Gaucher disease type 1 is important toprevent certain complications.