Gaucher Disease Treatment
- What It Is
- Risk Factors
- Living With
- User Questions
- Alternative Treatment
- Care Guide
- Questions for Your Doctor
- When to Contact a Doctor
- Find a Doctor
- Resource Guide
How to Treat Gaucher Disease
There is no treatment for the severe neurologic symptoms that may occur with type II and type III Gaucher. However, new treatment options for type I Gaucher include:
- Enzyme replacement therapy-This consists of a regular infusion with an enyzme replacement medicine. Examples of these medicines include alglucerase (derived from human cells), velaglucerase alfa (derived from human cells), and imiglucerase (chemically-modified enzyme). This therapy can help reduce skeletal abnormalities, liver and spleen size, and reverse some abnormal blood counts.
- Substrate reduction therapy-Miglustat (Zavesca) has been approved by the US Food and Drug Administration for treatment of type I Gaucher disease in adults who cannot receive hormone replacement therapy.
- Bone marrow transplant-A transplant is used only in patients with severe clinical symptoms and bone abnormalities. If it is successful, it provides a lifelong cure.
- Splenectomy -The surgical removal of the spleen may be done if enzyme replacement therapy is not available.
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