[HTML][HTML] Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome
J Muenzer, M Beck, CM Eng, R Giugliani, P Harmatz… - Genetics in …, 2011 - Elsevier
Purpose This study evaluated the safety and effectiveness of long-term enzyme replacement
therapy with idursulfase (recombinant human iduronate-2-sulfatase) in patients with Hunter
syndrome. Methods All 94 patients who completed a 53-week double-blinded study of
idursulfase enrolled in this open-labeled extension study and received intravenous
idursulfase at a dose of 0.5 mg/kg weekly for 2 years, and clinical outcomes and safety were
assessed. Results No change in percent predicted forced vital capacity was seen, but …
therapy with idursulfase (recombinant human iduronate-2-sulfatase) in patients with Hunter
syndrome. Methods All 94 patients who completed a 53-week double-blinded study of
idursulfase enrolled in this open-labeled extension study and received intravenous
idursulfase at a dose of 0.5 mg/kg weekly for 2 years, and clinical outcomes and safety were
assessed. Results No change in percent predicted forced vital capacity was seen, but …
