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Enzyme replacement therapy
| Enzyme replacement therapy | |
|---|---|
| Other names | ERT |
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]
- ^ a b c d e Ries, M (2017). "Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923–2016)". Journal of Inherited Metabolic Disease. 40 (3): 343–356. doi:10.1007/s10545-017-0032-8. PMID 28314976. S2CID 31320877.
- ^ Booth, C; Hershfield, M; Notarangelo, L; Buckley, R; Hoenig, M; Mahlaoui, N; Cavazzana-Calvo, M; Aiuti, A; Gaspar, H. B. (2007). "Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006)". Clinical Immunology. 123 (2): 139–47. doi:10.1016/j.clim.2006.12.009. PMID 17300989.
- ^ Ries, M (2017). "Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923–2016)". Journal of Inherited Metabolic Disease. 40 (3): 343–356. doi:10.1007/s10545-017-0032-8. PMID 28314976. S2CID 31320877. [verification needed]
- ^ Ries, M (2017). "Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923–2016)". Journal of Inherited Metabolic Disease. 40 (3): 343–356. doi:10.1007/s10545-017-0032-8. PMID 28314976. S2CID 31320877. [verification needed]
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علاج إنزيمي تعويضي Arabic Teràpia de reemplaçament enzimàtic Catalan Enzymersatztherapie German درمان جایگزینی با آنزیم FA Terapia di sostituzione enzimatica Italian 酵素補充療法 Japanese Enzymatyczna terapia zastępcza Polish Terapia de Reposição Enzimática Portuguese Ензимска супституциона терапија Serbian
