Intervention Protocol

Enzyme replacement and substrate reduction therapy for Gaucher disease

  1. Elad Shemesh1,*,
  2. Laura Deroma2,
  3. Bruno Bembi2,
  4. Patrick Deegan3,
  5. Carla Hollak4,
  6. Neal J Weinreb5,
  7. Timothy M Cox6

Editorial Group: Cochrane Cystic Fibrosis and Genetic Disorders Group

Published Online: 31 JAN 2013

Assessed as up-to-date: 14 NOV 2012

DOI: 10.1002/14651858.CD010324


How to Cite

Shemesh E, Deroma L, Bembi B, Deegan P, Hollak C, Weinreb NJ, Cox TM. Enzyme replacement and substrate reduction therapy for Gaucher disease (Protocol). Cochrane Database of Systematic Reviews 2013, Issue 1. Art. No.: CD010324. DOI: 10.1002/14651858.CD010324.

Author Information

  1. 1

    Ben-Gurion University of the Negev, Department of Clinical Biochemistry, Beer Sheva, Israel

  2. 2

    University Hospital "Santa Maria della Misericordia", Regional Co-ordinatior Center for Rare Diseases, Udine, Italy

  3. 3

    Addenbrookes Hospital, Lysosomal Disorders Unit, Cambridge, UK

  4. 4

    Academic Medical Center, Department of Internal Medicine, Division of Endocrinology & Metabolism, Amsterdam, Netherlands

  5. 5

    Northwest Oncology Hematology Associates PA, University Research Foundation for Lysosomal Storage Diseases, Coral Springs, Florida, USA

  6. 6

    Addenbrooke's Hospital (Box 157), Department of Medicine, Cambridge, Cambridgeshire, UK

*Elad Shemesh, Department of Clinical Biochemistry, Ben-Gurion University of the Negev, Beer Sheva, 84103, Israel. elad234@gmail.com.

Publication History

  1. Publication Status: New
  2. Published Online: 31 JAN 2013

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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:

  1. To assess the effectiveness and safety of ERTs and SRTs.
  2. To identify knowledge gaps which may result in new areas of research.