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

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...

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Bibliographic Details
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
Format: Article
Language:English
Published: NIHR Journals Library 2012-10-01
Series:Health Technology Assessment
Subjects:
effectiveness
cost-effectiveness
enzyme replacement therapy
substrate reduction therapy
lysosomal storage disorders
Online Access:https://doi.org/10.3310/hta16390

Internet

https://doi.org/10.3310/hta16390

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