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Results 1-10 of 29 (Search time: 0.009 seconds).
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PreviewIssue DateTitleAuthor(s)
2010Enzyme replacement therapy for mucopolysaccharidosis VI from 8 weeks of age - a sibling control studyMcGill, J.; Inwood, A.; Coman, D.; Lipke, M.; de Lore, D.; Swiedler, S.; Hopwood, J.
2010Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfataseHarmatz, P.; Yu, Z.; Giugliani, R.; Schwartz, I.; Guffon, N.; Teles, E.; Miranda, C.; Wraith, J.; Beck, M.; Arash, L.; Scarpa, M.; Ketteridge, D.; Hopwood, J.; Plecko, B.; Steiner, R.; Whitley, C.; Kaplan, P.; Swiedler, S.; Hardy, K.; Berger, K.; et al.
2011Screening patients referred to a metabolic clinic for lysosomal storage disordersFuller, M.; Tucker, J.; Lang, D.; Dean, C.; Fietz, M.; Meikle, P.; Hopwood, J.
2010Functional analysis of the HGSNAT gene in patients with Mucopolysaccharidosis IIIC (Sanfilippo C Syndrome)Fedele, A.; Hopwood, J.
2013Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapyBraunlin, E.; Rosenfeld, H.; Kampmann, C.; Johnson, J.; Beck, M.; Giugliani, R.; Guffon, N.; Ketteridge, D.; Miranda, C.; Scarpa, M.; Schwartz, I.; Teles, E.; Wraith, J.; Barrios, P.; Dias da Silva, E.; Kurio, G.; Richardson, M.; Gildengorin, G.; Hopwood, J.; Imperiale, M.; et al.
2012Helper-dependent canine adenovirus vector-mediated transgene expression in a neurodegenerative lysosomal storage disorderLau, A.; Rozaklis, T.; Ibanes, S.; Luck, A.; Beard, H.; Hassiotis, S.; Mazouni, K.; Hopwood, J.; Kremer, E.; Hemsley, K.
2010Allogeneic stem cell transplantation does not improve neurological deficits in mucopolysaccharidosis type IIIA miceLau, A.; Hannouche, H.; Rozaklis, T.; Hassiotis, S.; Hopwood, J.; Hemsley, K.
2010SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectorsLau, A.; Hopwood, J.; Kremer, E.; Hemsley, K.
2011Impact of high-dose, chemically modified sulfamidase on pathology in a murine model of MPS IIIARozaklis, T.; Beard, H.; Hassiotis, S.; Garcia, A.; Tonini, M.; Luck, A.; Pan, J.; Lamsa, J.; Hopwood, J.; Hemsley, K.
2013Neonatal bone marrow transplantation in MPS IIIA miceLau, A.; Shamsani, N.; Winner, L.; Hassiotis, S.; King, B.; Hopwood, J.; Hemsley, K.