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

Abstract 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 ar...

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Egile Nagusiak:	Tim A. Kanters, Ans T. van der Ploeg, Michelle E. Kruijshaar, Dimitris Rizopoulos, W. Ken Redekop, Maureen P. M. H. Rutten-van Mӧlken, Leona Hakkaart-van Roijen
Formatua:	Artigo
Hizkuntza:	Inglês
Argitaratua:	BMC 2017-12-01
Saila:	Orphanet Journal of Rare Diseases
Gaiak:	Pompe disease Enzyme replacement therapy Orphan drug Cost-effectiveness QALY
Sarrera elektronikoa:	http://link.springer.com/article/10.1186/s13023-017-0731-0
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