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

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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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: BMC 2017-12-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Pompe disease
Enzyme replacement therapy
Orphan drug
Cost-effectiveness
QALY
Online Access:http://link.springer.com/article/10.1186/s13023-017-0731-0

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http://link.springer.com/article/10.1186/s13023-017-0731-0

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