The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders
Objectives: To determine natural history and estimate effectiveness and cost of enzyme replacement therapy (ERT) and substrate replacement therapy (SRT) for patients with Gaucher disease, Fabry disease, mucopolysaccharidosis type I (MPS I), mucopolysaccharidosis type II (MPS II), Pompe disease and N...
Main 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 |
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Format: | Article |
Language: | English |
Published: |
NIHR Journals Library
2012-10-01
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Series: | Health Technology Assessment |
Subjects: | |
Online Access: | https://doi.org/10.3310/hta16390 |
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