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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Detalles Bibliográficos
Autores principales: Kanters, Tim A, Hoogenboom-Plug, Iris, Rutten-Van Mölken, Maureen PMH, Redekop, W Ken, van der Ploeg, Ans T, Hakkaart, Leona
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2014
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4038090/
https://www.ncbi.nlm.nih.gov/pubmed/24884717
http://dx.doi.org/10.1186/1750-1172-9-75

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4038090/
https://www.ncbi.nlm.nih.gov/pubmed/24884717
http://dx.doi.org/10.1186/1750-1172-9-75

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