Enzyme Replacement Therapy for the Management of the Mucopolysaccharidoses

Overview

Journal Int J Clin Pharmacol Ther

Specialty Pharmacology

Date 2009 Dec 31

PMID 20040314

Citations 9

Authors

J E Wraith

Affiliations

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Abstract

Enzyme replacement therapy (ERT) is now available for several of the mucopolysaccharidosis disorders. This brief review summarizes the role of ERT in reducing the burden of peripheral disease in many patients with mucopolysaccharidosis disorders, and describes the challenges that remain in treating the neurological manifestations of these conditions.

Citing Articles

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Tkachyova I, Fan X, Lamhonwah A, Fedyshyn B, Tein I, Mahuran D PLoS One. 2016; 11(9):e0162145.

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Brands M, Gungor D, van den Hout J, Karstens F, Oussoren E, Plug I J Inherit Metab Dis. 2014; 38(2):323-31.

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