Browsing Biological Sciences by Author "Whiteman, David A.H."
Now showing items 1-2 of 2
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Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients
Giugliani, Roberto; Harmatz, Paul; Jones, Simon A.; Mendelsohn, Nancy J.; Vellodi, Ashok; Qiu, Yongchang; Hendriksz, Christian J.; Vijayaraghavan, Suresh; Whiteman, David A.H.; Pano, Arian (2017) [Journal article]Objectives: This 109-week, nonrandomized, observational study ofmucopolysaccharidosis II (MPS II) patients already enrolled in the Hunter Outcome Survey (HOS) (NCT00882921), assessed the long-termimmunogenicity of idursulfase, ... -
Ten years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registry
Muenzer, Joseph; Jones, Simon A.; Tylki-Szymanska, Anna; Harmatz, Paul; Mendelsohn, Nancy J.; Guffon, Nathalie; Giugliani, Roberto; Burton, Barbara K.; Scarpa, Maurizio; Beck, Michael; Jangelind, Yvonne; Hernberg-Stahl, E.; Larsen, Maria Paabøl; Pulles, Tom; Whiteman, David A.H. (2017) [Journal article]Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare lysosomal storage disease with progressive multisystem manifestations caused by deficient activity of the enzyme iduronate-2-sulfatase. ...