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Assessing the Value for Money of Enzyme Replacement Therapy in Gaucher Disease Types 1 and 3b: Can Expanded Coverage Be Justified?
Abstract
Background and Objectives
The Health Intervention and Technology Assessment Program was commissioned to conduct a cost–utility and budget impact analysis of enzyme replacement therapy (ERT) for Gaucher disease types 1 and 3b. The findings from this assessment are to support the decision-m
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