Health Technology Assessment

The effectiveness and cost of enzyme replacement and substrate reduction therapies: a longitudinal cohort study of people with lysosomal storage disorders

  • Type:
    Extended Research Article Our publication formats
  • Headline:
    Study provides further evidence on the effectiveness of enzyme replacement therapy in people with lysosomal storage disorders.
  • Authors:
    K Wyatt,
    W Henley,
    L Anderson,
    R Anderson,
    V Nikolaou,
    K Stein,
    L Klinger,
    D Hughes,
    S Waldek,
    R Lachmann,
    A Mehta,
    A Vellodi,
    S Logan
    Detailed Author information

    K Wyatt1, W Henley1, L Anderson1, R Anderson1, V Nikolaou1, K Stein1, L Klinger1, D Hughes2, S Waldek3, R Lachmann4, A Mehta2, A Vellodi5, S Logan1,*

    • 1 Peninsula Medical School, University of Exeter, Exeter, UK
    • 2 Department of Haematology, Royal Free Campus, University College, London, UK
    • 3 Retired Metabolic Physician, formerly of Salford Royal NHS Foundation Trust, Salford, UK
    • 4 University College London Hospitals, London, UK
    • 5 Great Ormond Street Hospital for Children NHS Trust, London, UK
  • Funding:
    Health Technology Assessment programme
  • Journal:
  • Issue:
    Volume: 16, Issue: 39
  • Published:
  • Citation:
    Primary research. Wyatt K, Henley W, Anderson L, Anderson R, Nikolaou V, Stein K, et al. Volume 16, number 39. Published October 2012. The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders. Health Technol Assess 2012;16(39). https://doi.org/10.3310/hta16390
  • DOI:
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