Enzyme replacement therapy

Enzyme replacement therapy is a medical treatment replacing an enzyme in patients in whom that particular enzyme is deficient or absent. Usually this is done by giving the patient an intravenous (IV) infusion containing the enzyme. Enzyme replacement therapy is currently available for some lysosomal diseases: Gaucher disease, Fabry disease, MPS I and Glycogen storage disease type II. Enzyme replacement therapy does not "treat" the underlying disease, only the symptoms.

Enzyme replacement therapy