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

BACKGROUND: Pompe disease is a rare, progressive, metabolic disease, and the first treatable inheritable muscle disorder. Enzyme replacement therapy (ERT) with alglucosidase alfa is disease specific and the only medicinal product authorized for the treatment of Pompe disease. Costs of ERT are very h...

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Vydáno v:Orphanet J Rare Dis
Hlavní autoři: Kanters, Tim A., van der Ploeg, Ans T., Kruijshaar, Michelle E., Rizopoulos, Dimitris, Redekop, W. Ken, Rutten-van Mӧlken, Maureen P. M. H., Hakkaart-van Roijen, Leona
Médium: Artigo
Jazyk:Inglês
Vydáno: BioMed Central 2017
Témata:
Research
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5729274/
https://ncbi.nlm.nih.gov/pubmed/29237491
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-017-0731-0
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