Specialty Pharmacy

Enzyme Replacement Therapy (ERT)

What is Enzyme Replacement Therapy (ERT)?

Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme. Enzyme replacement therapy (ERT) is commonly used to treat patients with lysosomal diseases.

ERT is currently available for some lysosomal storage diseases:

  • Gaucher disease
  • Fabry disease
  • MPS I
  • MPS II (Hunter syndrome)
  • MPS VI
  • Pompe disease

ERT does not correct the underlying genetic defect, but it increases the concentration of the enzyme that the patient is lacking. ERT has also been used to treat patients with severe combined immunodeficiency (SCID), resulting from an adenosine deaminase deficiency (ADA-SCID). Patients receive ERT via IV infusion, which usually takes 1 to 2 hours. Patients typically need an ERT infusion every 2 weeks, depending on the individual.