0000000000597812
AUTHOR
Akemi Tanaka
PO-0096 Clinical Effectiveness Of Idursulfase In Boys Aged 0–5 Years With Hunter Syndrome: 3-year Data From The Hunter Outcome Survey
Background and aims Symptoms of Hunter syndrome typically become apparent at 2–4 years of age. Previous analyses have demonstrated improvements in certain clinical measures in young patients receiving idursulfase (Shire); however, data on long-term idursulfase use in these patients remain limited. This analysis used data available in the Hunter Outcome Survey (HOS), a global, observational registry sponsored by Shire, to investigate long-term effectiveness of idursulfase in boys with Hunter syndrome aged 0–5 years. Methods As of January 2014, 260/564 males followed prospectively in HOS had received ≥1 idursulfase infusion (excluding those who had received a bone marrow transplant or were en…
Clinical course of sly syndrome (mucopolysaccharidosis type VII).
WOS: 000377110800007