Enzyme replacement therapy for the management of the mucopolysaccharidoses

J. E. Wraith

    Research output: Contribution to journalArticlepeer-review

    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. ©2009 Dustri-Verlag Dr. K. Feistle.
    Original languageEnglish
    Pages (from-to)S63-S65
    JournalInternational Journal of Clinical Pharmacology and Therapeutics
    Volume47
    Issue number1
    DOIs
    Publication statusPublished - 2009

    Keywords

    • Enzyme replacement therapy
    • Hematopoietic stem cell therapy
    • Mucopolysaccharidosis disorders
    • Palliative care
    • Surgery

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