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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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2017
Assuntos:
Research
Acesso em linha: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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