Definitions for "Enzyme replacement therapy"
A form of treatment used for some LSDs in which patients get an intravenous infusion to replace the missing or faulty enzyme causing the LSD. exon: Coding sequence of DNA present in mature messenger RNA
A method of treating genetic disorders which are due to a deficiency of a particular enzyme. Overcoming the deficiency by providing the body with the enzyme enables the cells to function correctly and the symptoms of the disorder may be corrected.
A therapy designed to address the underlying enzyme deficiency associated with a lysosomal storage disorder or other disease characterized by an enzyme deficiency.