Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease

BACKGROUND: Infantile Pompe disease is a rare metabolic disease. Patients generally do not survive the first year of life. Enzyme replacement therapy (ERT) has proven to have substantial effects on survival in infantile Pompe disease. However, the costs of therapy are very high. In this paper, we as...

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Bibliographic Details
Main Authors: Kanters, Tim A, Hoogenboom-Plug, Iris, Rutten-Van Mölken, Maureen PMH, Redekop, W Ken, van der Ploeg, Ans T, Hakkaart, Leona
Format: Artigo
Language:Inglês
Published: BioMed Central 2014
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Research
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4038090/
https://ncbi.nlm.nih.gov/pubmed/24884717
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-9-75
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