Cost-effectiveness of enzyme replacement therapy for Fabry disease

  title={Cost-effectiveness of enzyme replacement therapy for Fabry disease},
  author={Saskia M. Rombach and Carla E. M. Hollak and Gabor E. Linthorst and Marcel G. W. Dijkgraaf},
  booktitle={Orphanet journal of rare diseases},
BACKGROUND The cost-effectiveness of enzyme replacement therapy (ERT) compared to standard medical care was evaluated in the Dutch cohort of patients with Fabry disease. METHODS Cost-effectiveness analysis was performed using a life-time state-transition model. Transition probabilities, effectiveness data and costs were derived from retrospective data and prospective follow-up of the Dutch study cohort consisting of males and females aged 5-78 years. Intervention with ERT (either agalsidase… CONTINUE READING
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