Enzyme replacement therapy
Medical treatment / From Wikipedia, the free encyclopedia
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Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body.[1] Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme.[1]
ERT is available for some lysosomal storage diseases: Gaucher disease, Fabry disease, MPS I, MPS II (Hunter syndrome), MPS VI and Pompe disease.[1] ERT does not correct the underlying genetic defect, but it increases the concentration of the enzyme that the patient is lacking.[1] ERT has also been used to treat patients with severe combined immunodeficiency (SCID) resulting from an adenosine deaminase deficiency (ADA-SCID).[2]
Other treatment options for patients with enzyme or protein deficiencies include substrate reduction therapy, gene therapy, and bone-marrow derived stem cell transplantation.[1][3][4]