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Intervention Protocol

Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I

  1. Elisabeth Jameson*,
  2. Simon Jones,
  3. James E Wraith

Editorial Group: Cochrane Cystic Fibrosis and Genetic Disorders Group

Published Online: 5 OCT 2011

Assessed as up-to-date: 25 AUG 2011

DOI: 10.1002/14651858.CD009354


How to Cite

Jameson E, Jones S, Wraith JE. Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I (Protocol). Cochrane Database of Systematic Reviews 2011, Issue 10. Art. No.: CD009354. DOI: 10.1002/14651858.CD009354.

Author Information

  1. 6th Floor, St Mary's Hospital, Biochemical Genetics Unit, Dept of Genetic Medicine, Manchester, UK

*Elisabeth Jameson, Biochemical Genetics Unit, Dept of Genetic Medicine, 6th Floor, St Mary's Hospital, Hathersage Road, Manchester, M13 9WL, UK. Elisabeth.Jameson@cmft.nhs.uk. elisabethjameson@hotmail.com.

Publication History

  1. Publication Status: Edited (no change to conclusions)
  2. Published Online: 5 OCT 2011

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Abstract

  1. Top of page
  2. Abstract

This is the protocol for a review and there is no abstract. The objectives are as follows:

The objectives of this review will be to assess the effectiveness and safety of ERT with laronidase in the management of patients with:

  1. MPS I who do not undergo HSCT;
  2. MPS I who receive ERT prior to HSCT.