Overview

Because it can affect the body in many different ways, Gaucher disease may require a number of different treatments. Many interventions for Type 1 Gaucher disease have historically been intended to ease various signs and symptoms. These treatments may include pain medications, surgery for bone and joint problems, and possibly removal of the spleen.

These measures still have a place in the management of Gaucher disease. However, in 1991, Gaucher disease management shifted with the advent of targeted enzyme replacement therapy, developed by Genzyme Corporation in cooperation with the NIH. With the introduction of Cerezyme^® (imiglucerase for injection) in 1994 and with its predecessor, clinicians have been able to address the disease process itself, and therefore alleviate and even reverse many effects of Type 1 Gaucher disease.

What does Cerezyme do?

In ordinary cells, an enzyme called glucocerebrosidase breaks down specific fat molecules that accumulate within a compartment of the cell. As discussed in the About Gaucher section, people with Gaucher disease lack this enzyme. When administered to people with Type 1 Gaucher disease, Cerezyme acts like the naturally occurring enzyme glucocerebrosidase to break down the fat molecules that have accumulated in Gaucher cells. Because of this activity, Cerezyme therapy is classified as enzyme replacement therapy (ERT).

Cerezyme therapy has been shown to help relieve or reverse many of the signs and symptoms of Type 1 Gaucher disease. However, treatment experiences may vary from one person to the next. To find out more about the possible effects of Cerezyme therapy, read What to Expect from Therapy.

For more information on treatment with Cerezyme therapy, see Enzyme Replacement Therapy, Full Product Information (PDF) or important safety information below.

Long-term treatment

Cerezyme therapy is not a cure for Gaucher disease; that is, it does not correct the underlying genetic defect. In order to continue to benefit from the treatment, symptomatic patients must receive intravenous infusions for the rest of their lives, even though they may feel better. That’s because, once therapy stops, Gaucher cells may build up again and symptoms may come back. Turn to Continuing Treatment to learn more.

Gaucher disease treatment centers

Gaucher disease treatment centers provide specialized treatment and counseling services for patients with Type 1 Gaucher disease. To find a facility in your area, go to Locate a Treatment Center.

Important Safety Information

Side effects related to Cerezyme® (imiglucerase for injection) administration have been reported in less than 15% of patients. Each of the following events occurred in less than 2% of the total patient population. Reported side effects include nausea, vomiting, abdominal pain, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and rapid heart rate. Because Cerezyme® therapy is administered by intravenous infusion, reactions at the site of injection may occur: discomfort, itching, burning, swelling or uninfected abscess. Symptoms suggestive of allergic reaction include anaphylactoid reaction (a serious allergic reaction), itching, flushing, hives, an accumulation of fluid under the skin, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), and low blood pressure. Approximately 15% of patients have developed immune responses (antibodies); periodic monitoring by your physician is suggested. Patients should notify their physician immediately if they experience any side effects with treatment. Cerezyme is available by prescription only. For more information, consult your physician. To learn more, please see the full product information (PDF) or contact Genzyme at 1-800-745-4447.