Review
doi: 10.5414/cpp47063.
Enzyme replacement therapy for the management of the mucopolysaccharidoses
Affiliations
- PMID: 20040314
- DOI: 10.5414/cpp47063
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Review
Enzyme replacement therapy for the management of the mucopolysaccharidoses
Int J Clin Pharmacol Ther.
2009.
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.
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