Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)

被引：0
|
作者：
Joseph Muenzer
Jaco Botha
Paul Harmatz
Roberto Giugliani
Christoph Kampmann
Barbara K. Burton
机构：
[1] University of North Carolina at Chapel Hill,Department of Genetics
[2] Takeda Pharmaceuticals International AG,undefined
[3] UCSF Benioff Children’s Hospital Oakland,undefined
[4] UFRGS,undefined
[5] Medical Genetics Service,undefined
[6] HCPA,undefined
[7] and INAGEMP,undefined
[8] Johannes Gutenberg University,undefined
[9] Ann & Robert H. Lurie Children’s Hospital of Chicago,undefined
来源：
Orphanet Journal of Rare Diseases | / 16卷
关键词：
Mucopolysaccharidosis II; MPS II; Hunter syndrome; Lysosomal storage disease; Statistical modeling; Disease registry; Idursulfase; Enzyme replacement therapy;
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