Enzyme Replacement Therapy is proving to be Beneficial for Patients Suffering fr

Posted by Sachin CMI on December 4th, 2020

Enzyme replacement therapy (ERT) is a medical treatment used to replace an enzyme that is deficient or absent in the body. ERT is mainly used in Fabry disease, Gaucher disease, Hunter syndrome, Pompe disease, MPS I, and MPS VI. Although ERT does not correct the underlying genetic effect defect, it increases the concentration of the enzyme that the patient is lacking. Furthermore, ERT is being used to treat patients suffering from severe combined immunodeficiency (SCID) resulting from an adenosine deficiency. In particular, enzyme replacement therapy is used in the treatment of lysosomal storage disease treatment.

ERT involves intravenous (IV) infusions so as to correct underlying enzyme deficiency, which leads to symptoms of Gaucher disease. The primary function of ERT is to balance low levels of glucocerebrosidase (GCase) enzyme with the help of modified version of the enzyme. The patients receive ERT via IV infusions, which generally takes up to 1-2 hours. The majority of patients receive ERT at infusion centers. The infusion centers are specialized medical facilities with expertise in IV infusions. Furthermore, patients may also receive ERT infusions at hospitals and physicians’ offices. This, in turn, allows medicine practitioners to keep track of the patient’s recovery and development. Some patients are given ERT infusions at home with the help of a home health nurse. This typically saves time and hassle of travel.

There certain advantages and disadvantages of enzyme replacement therapy. The advantages include fewer side effects as compared to substrate reduction therapy (SRT), longer drug history, and wider availability. On the contrary, the disadvantages include inconvenience, more invasive than SRT, and fatigue. Side effects of ERT include rash, fever, flushed skin, headache, chest discomfort, high or low blood pressure, muscle tremors, nausea & vomiting, dizziness, and quickened heart rate.

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