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



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


Vol 9: Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease. - Download this document for free, or read online. Document in PDF available to download.

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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

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

Source: https://archive.org/







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