Enzyme replacement therapy (ERT)

Enzyme replacement therapy (ERT) is a type of medical treatment that involves replacing an enzyme that is missing or not working properly in the body. It is most commonly used to treat a variety of rare genetic and metabolic disorders, such as Gaucher disease, Fabry disease, and Pompe disease. In ERT, a specific enzyme is administered either by intravenous (IV) infusion or subcutaneous (SC) injection to replace the missing or non-functioning enzyme. The enzyme is typically a recombinant form of the protein, meaning it is produced in a lab using genetic engineering. The goal of ERT is to restore normal enzyme activity and reduce symptoms. ERT can also help slow down disease progression and improve quality of life.