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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Detaylı Bibliyografya
Asıl Yazarlar: Kanters, Tim A, Hoogenboom-Plug, Iris, Rutten-Van Mölken, Maureen PMH, Redekop, W Ken, van der Ploeg, Ans T, Hakkaart, Leona
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: BioMed Central 2014
Konular:
Research
Online Erişim: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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