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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5729274/
https://www.ncbi.nlm.nih.gov/pubmed/29237491
http://dx.doi.org/10.1186/s13023-017-0731-0

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5729274/
https://www.ncbi.nlm.nih.gov/pubmed/29237491
http://dx.doi.org/10.1186/s13023-017-0731-0

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