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Enzyme Replacement Therapy (ERT) FDA approved in 1991

In 1991, Gaucher disease management shifted with the advent of targeted enzyme replacement therapy (ERT), 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. People with Gaucher disease lack this enzyme. (See "About Gaucher disease" at 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.

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. (Information obtained from