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

被引：0
作者：
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
机构：
[1] Erasmus University Rotterdam,Institute for Medical Technology Assessment
[2] Center for Lysosomal and Metabolic Diseases,undefined
[3] Department of Pediatrics Sophia’s Children’s Hospital,undefined
[4] Department of Biostatistics,undefined
[5] Erasmus University Medical Center,undefined
[6] Erasmus School of Health Policy & Management,undefined
[7] Erasmus University Rotterdam,undefined
来源：
Orphanet Journal of Rare Diseases | / 12卷
关键词：
Pompe disease; Enzyme replacement therapy; Orphan drug; Cost-effectiveness; QALY;
D O I：
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