Name: Enzyme replacement therapy for the management of the mucopolysaccharidoses
Creator: Wraith, J. E.
Keywords: 0301 basic medicine, Mucopolysaccharidosis VI, N-Acetylgalactosamine-4-Sulfatase, Mucopolysaccharidosis I, Hematopoietic Stem Cell Transplantation, Iduronate Sulfatase, Combined Modality Therapy, Recombinant Proteins, 3. Good health, Iduronidase

descriptionPublicationkeyboard_double_arrow_right Article 23 Dec 2009 English Publisher:Dustri-Verlgag Dr. Karl FeistleJournal:Int. Journal of Clinical Pharmacology and Therapeutics (issn: 0946-1965,

Authors: Wraith, J. E.;

doi: 10.5414/cpp47063

pmid: 20040314

Enzyme replacement therapy for the management of the mucopolysaccharidoses

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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.

Related Organizations

University of Salford
United Kingdom
Boston Children's Hospital
United States
Manchester University NHS Foundation Trust
United Kingdom
Royal Manchester Children's Hospital
United Kingdom

Keywords

Mucopolysaccharidosis VI, N-Acetylgalactosamine-4-Sulfatase, Mucopolysaccharidosis I, Hematopoietic Stem Cell Transplantation, Iduronate Sulfatase, Combined Modality Therapy, Recombinant Proteins, Iduronidase, Enzyme replacement therapy, Hematopoietic stem cell therapy, Palliative care, Humans, Surgery, Enzyme Replacement Therapy, Mucopolysaccharidosis disorders, Mucopolysaccharidosis II

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