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

@inproceedings{Giugliani2014AMO,
  title={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},
  author={Roberto Giugliani and W L Hwu and Anna Tylki-Szymańska and David A.H. Whiteman and Arian Pano},
  booktitle={Genetics in Medicine},
  year={2014}
}
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 adverse events, anti-idursulfase antibodies, vital signs, physical examination, 12-lead electrocardiogram, concomitant medications or procedures, and laboratory testing (clinical chemistry, hematology… CONTINUE READING
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