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

@inproceedings{Wyatt2013TheEA,
  title={The effectiveness and cost of enzyme replacement and substrate reduction therapies : a longitudinal cohort study of people with lysosomal storage disorders},
  author={Katrina Mary Wyatt and William Edward Henley and Laura Anderson and Rosie Anderson and Vassilis Nikolaou and Kari Stein and Lisa Klinger and Doriann Hughes and Stephen Waldek and Ricarda Lachmann and Akshay Mehta and Ashok Vellodi and Stephanie Logan},
  year={2013}
}
List of abbreviations Executive summary Background Objectives Methods Results Conclusions Recommendations for research Funding Chapter 1 Background Lysosomal storage disorders Summary of lysosomal storage disorders investigated in this study Patterns of treatment in England Rationale for the cohort study Background and objectives Chapter 2 Methods Study procedures Data collection Methods used in analysis Key for longitudinal model outputs Cost data analysis methods Changes to original protocol… CONTINUE READING
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Lysosomal defects and storage

  • FM Platt, SU Walkley
  • 2004
Highly Influential
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