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Additional file 1 of 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)

, 2021
Additional file 1. Table S1 Summary of demographics and clinical characteristics for patients included in at least one statistical model. Table S2 Comparison of results from two modeling approaches to evaluate palpable liver size over time following IV ...
Paul Harmatz (426492), Barbara K. Burton (3849295), Joseph Muenzer (11627733), Jaco Botha (2792269), Roberto Giugliani (398540), Christoph Kampmann (3112212)   +5 more
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Additional file 1 of Sustained clinical benefit of idursulfase beta in mucopolysaccharidosis II: two-year experience from a phase 3 extension study including patients switched from idursulfase

Supplementary Material 1: The following supplementary information can be found online. Tables S1 to S3: Safety, efficacy, and immunogenicity results in the one patient enrolled only in the extension study. Table S4: Incidence of adverse drug reactions. Table S5: Serious adverse events reported during the whole study period.
Minji Im, Ari Song, Hyojung Park, Jiyeon Kim, Chiwoo Kim, Eu-Seon Noh, Ga young Bae, Juyoung Sung, JiHoon Hwang, Yoon Ji Ahn, Haein Kim, Youngsin Oh, Bogyeong Kim, Sung Yoon Cho   +13 more
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Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future

, 2017
David AH Whiteman,* Alan Kimura* Research & Development, Shire Human Genetic Therapies, Inc., Lexington, MA, USA *These authors contributed equally to this work Abstract: Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is
Whiteman DAH, Kimura A
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Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey

, 2010
To use our experience with patients in the observational database HOS - the Hunter Outcome Survey - to evaluate the feasibility of home infusions of idursulfase for patients with mucopolysaccharidosis type II (MPS II)
Barbara K. Burton, Jones, SA, Burton, BK, GUFFON N, VAN DER PLOEG AT, Jane Roberts, van der Ploeg, Ans, SCARPA, MAURIZIO, BURTON BK, Guffon, N, Simon A. Jones, Feliciani, Claudio, ROBERTS J, Nathalie Guffon, Ans T. van der Ploeg, JONES SA, Roberts, J, van, der Ploeg AT, Van Der Ploeg, At   +18 more
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Survival in idursulfase-treated and untreated patients with mucopolysaccharidosis type II:data from the Hunter Outcome Survey (HOS) [PDF]

, 2017
Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a life-limiting, multisystemic disease with varying presentation and severity.
Jones, Simon A., Barbara K. Burton, Simon A. Jones, Jego, Virginie, Jaromir Mikl, Burton, Barbara K., Mikl, Jaromir, Virginie Jego   +7 more
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Russian doctors have mastered intracerebroventricular administration of idursulfase beta

Педиатрическая фармакология
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article Editorial
doaj  

A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4-7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy

, 2017
Purpose: The primary objective of this study was to determine the safety of idursulfase in Hunter syndrome patients aged 5 years or younger. ;Methods: Idursulfase (0.5 mg/kg) was administered intravenously on a weekly basis (52 infusions per patient) in ...
Giugliani, Roberto;Hwu, Wuh-Liang;Tylki-Szymanska, Anna;Whiteman, David A. H.;Pano, Arian, 胡務亮   +1 more
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PRO40 BUDGET IMPACT ANALYSIS OF IDURSULFASE BETA IN THE TREATMENT MUCOPOLYSACCHARIDOSIS TYPE II IN THE RUSSIAN FEDERATION [PDF]

Value in Health, 2019
R. Yagudina, A. Kulikov, V. Serpik, E. Kostina   +3 more
openaire   +1 more source

Phi Beta Kappa, Alpha Chapter Records [PDF]

, 1875
Phi Beta Kappa was founded by five students at the College of William and Mary in 1776. This minute book documents the organization in the period 1875-1905.
Phi Beta Kappa, College of William and Mary. Students   +1 more
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Successful desensitization to idursulfase in a patient with type II mucopolysaccharidosis (Hunter syndrome).

, 2011
Hunter syndrome or type II mucopolysaccharidosis is a rare lysosomal storage disease of X-linked recessive inheritance. It is characterized by a lack of the enzyme iduronate 2 sulfatase (I2S), which leads to the accumulation of glycosaminoglycans in many
Gomez, J F, Serrano Reyes, Carlos Daniel
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