My Community - Clinical Information


How can Gaucher disease be managed?

You can take an active role in managing Gaucher disease and the effects it has on your life. Living a healthy lifestyle can help. This includes having a healthy diet (eating a variety of fresh foods, such as vegetables, fruits, and grains), not smoking, and getting regular exercise as recommended by your doctor. Just like everyone else, people with Gaucher disease should have regular medical checkups and screening tests. People with Gaucher disease need bone checkups and physical therapy. Sometimes they need special medicines to strengthen their bones.

A highly experienced international group of doctors who specializes in treating people with Gaucher disease recommends enzyme replacement therapy with Cerezyme as the standard of care treatment for people with Gaucher disease. Cerezyme has been available in Canada since 1997. It has been used to treat more than 5000* people around the world.

What is Cerezyme (imiglucerase)?

Cerezyme is a modified form of the human enzyme glucocerebrosidase that works by replacing the missing or nonworking enzyme, or enzyme with minimal activity, with a working enzyme. Cerezyme is a form of the enzyme that people with Gaucher disease need. Cerezyme is produced using genetic engineering technology (also called recombinant DNA technology) to make large amounts of the needed enzyme. Produced in a highly specialized laboratory, the enzyme is changed and made safe so that human Gaucher cells can use it.

How does Cerezyme work?

Cerezyme acts like the enzyme that is produced naturally in people who do not have Gaucher disease. It helps to break down the fatty substance that builds up inside the Gaucher cells in people with the disease. These cells start to shrink and disappear. Cerezyme does not fix the abnormal gene that causes the disease. To have better health, you must receive your treatment regularly as scheduled by your doctor.

Cerezyme reverses or controls many of the signs and symptoms of Gaucher disease, including:

  • Anemia (low red blood cell count)
  • Low platelet count (platelets help to stop bleeding)
  • Enlarged spleen
  • Enlarged liver
  • Bone disease

Questions about Cerezyme

How is Cerezyme given?

Cerezyme is given by intravenous (IV) infusion usually every two weeks. An infusion is when fluid or medicine is put into a vein with a needle or through a port. A port is a small device placed under the skin that allows treatment to be given with or without a needle depending on the type of port used. A port is used when it continues to be hard to find a vein to inject.

Why does Cerezyme need to be continued?

As you feel better, you may wonder why you still need Cerezyme. You may feel tempted to take a break or to stop treatment completely. Keep in mind that when Cerezyme stops, the signs and symptoms of the disease can come back. Then, the disease might damage your body in ways that cannot be helped when Cerezyme is restarted. To ensure you have better health, you need to commit to giving your body what it needs — regular infusions with the missing enzyme.

What happens if you miss a dose of Cerezyme?

If you miss a dose of Cerezyme, talk with your doctor about making it up. As long as the total amount of Cerezyme you take in a month does not change, a single missed dose can be corrected and is not likely to cause harm.

What happens if you stop Cerezyme?

Some people may want to take a break from treatment. But this is not recommended. Once you have met your treatment goals, it is important to continue with treatment. This will help you to maintain your improved health over time. If treatment stops, signs and symptoms of the disease may return. Your platelet and red blood cell counts can drop. Fatigue and bone pain may return. The risk for bone complications rises again. Your liver and spleen may become enlarged once again. Any gains you may have made in your health and feeling better while taking Cerezyme may be lost if you stop your treatment. Cerezyme is not a cure for Gaucher disease. If you have the disease, you must have intravenous infusions for the rest of your life, even if you feel better.

To find out more about Cerezyme read the Product Monograph.

Read more clinical information about Gaucher disease in Publications and Sanofi Genzyme Gaucher Research.