Results 61 to 70 of about 741,582 (71)

A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II [PDF]

Genetics in Medicine, 2016
Approximately two-thirds of patients with the lysosomal storage disease mucopolysaccharidosis II have progressive cognitive impairment. Intravenous (i.v.) enzyme replacement therapy does not affect cognitive impairment because recombinant iduronate-2 ...
Luying Pan, Joseph Muenzer, Christian Hendriksz   +2 more
exaly   +2 more sources

Intravenous Idursulfase for the Treatment of Mucopolysaccharidosis Type II: A Systematic Literature Review

International Journal of Molecular Sciences
Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare, X-linked disorder caused by deficient activity of the enzyme iduronate-2-sulfatase. Signs and symptoms typically emerge at 1.5–4 years of age and may include cognitive impairment ...
David Whiteman, Walla Al-Hertani
exaly   +2 more sources

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PRO40 BUDGET IMPACT ANALYSIS OF IDURSULFASE BETA IN THE TREATMENT MUCOPOLYSACCHARIDOSIS TYPE II IN THE RUSSIAN FEDERATION

Value in Health, 2019
Vyacheslav Serpik, Andrey Kulikov, R Yagudina   +2 more
exaly  

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

Genetics in Medicine, 2011
David Whiteman, Roberto Giugliani, Joseph Muenzer   +2 more
exaly  

Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II

Molecular Genetics and Metabolism, 2007
Joseph Muenzer, Jeffrey M Dacosta
exaly  

Idursulfase treatment of Hunter syndrome in children younger than 6 years: Results from the Hunter Outcome Survey

Genetics in Medicine, 2011
Anna Tylki-Szymanska, Roberto Giugliani, Joseph Muenzer   +2 more
exaly  

Correlation between urinary GAG and anti-idursulfase ERT neutralizing antibodies during treatment with NICIT immune tolerance regimen: A case report

Molecular Genetics and Metabolism, 2017
Sarah Kim-Mcolash
exaly  

Site-specific characterization of mannose-6-phosphate-containing N-glycans on recombinant idursulfase beta for lysosomal targeting in Hunter syndrome therapy

International Journal of Biological Macromolecules
Jeongeun Kim
exaly  

Incidence and timing of infusion-related reactions in patients with mucopolysaccharidosis type II (Hunter syndrome) on idursulfase therapy in the real-world setting: A perspective from the Hunter Outcome Survey (HOS)

Molecular Genetics and Metabolism, 2011
David Whiteman
exaly  

Comparative study of idursulfase beta and idursulfase in vitro and in vivo

Journal of Human Genetics, 2016
exaly