Browsing by Author "Burton, Barbara K."
Now showing items 1-2 of 2
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Evaluation of the long-term treatment efects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling : data from the Hunter Outcome Survey (HOS)
Muenzer, Joseph; Botha, Jaco; Harmatz, Paul; Giugliani, Roberto; Kampmann, Christoph; Burton, Barbara K. (2021) [Journal article]Background: Mucopolysaccharidosis II (MPS II; Hunter syndrome) is a rare, life-limiting lysosomal storage disease caused by defcient iduronate-2-sulfatase activity. Enzyme replacement therapy (ERT) with intravenous (IV) ... -
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. ...