Vol 9: Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease.Reportar como inadecuado



 Vol 9: Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease.


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This article is from Orphanet Journal of Rare Diseases, volume 9.AbstractBackground: 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 hig

Autor: Kanters, Tim A; Hoogenboom-Plug, Iris; Rutten-Van Molken, Maureen PMH; Redekop, W Ken; van der Ploeg, Ans T; Hakkaart, Leona

Fuente: https://archive.org/



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