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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

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 an open-label study. Safety monitoring included...

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書誌詳細
主要な著者: Giugliani, Roberto, Hwu, Wuh-Liang, Tylki-Szymanska, Anna, Whiteman, David A.H., Pano, Arian
フォーマット: Artigo
言語:Inglês
出版事項: Nature Publishing Group 2014
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC4052427/
https://ncbi.nlm.nih.gov/pubmed/24202085
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gim.2013.162
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