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Issue Date
Title
Author(s)
2013
Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy
Braunlin, 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.
2005
Laronidase treatment of mucopolysaccharidosis I
Wraith, J.
;
Hopwood, J.
;
Fuller, M.
;
Meikle, P.
;
Brooks, D.
2005
Pharmacokinetic profile of recombinant human N-acetylgalactosamine 4-sulphatase enzyme replacement therapy in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): a phase I/II study
Harmatz, P.
;
Kramer, W.
;
Hopwood, J.
;
Simon, J.
;
Butensky, E.
;
Swiedler, S.
1996
Identification, expression, and biochemical characterization of N-acetylgalactosamine-4-sulfatase mutations and relationship with clinical phenotype in MPS-VI patients
Litjens, T.
;
Brooks, D.
;
Peters, C.
;
Gibson, G.
;
Hopwood, J.
2008
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase
Harmatz, P.
;
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.
;
Yu, Z.
;
Swiedler, S.
;
Decker, C.
2015
Butanolysis derivatization: improved sensitivity in LC-MS/MS quantitation of heparan sulfate in urine from mucopolysaccharidosis patients
Trim, P.
;
Hopwood, J.
;
Snel, M.
1997
Human mucopolysaccharidosis IIID: clinical, biochemical, morphological and immunohistochemical characteristics
Jones, M.
;
Alroy, J.
;
Rutledge, J.
;
Taylor, J.
;
Alvord Jnr., E.
;
Toone, J.
;
Applegarth, D.
;
Hopwood, J.
;
Skutelsky, E.
;
Ianelli, C.
;
Thorley Lawson, D.
;
Mitchell Herpolsheimer, C.
;
Arias, A.
;
Sharp, P.
;
Evans, W.
;
Sillence, D.
;
Cavanagh, K.
2002
Correlation among genotype, phenotype, and biochemical markers in Gaucher disease: Implications for the prediction of disease severity
Whitfield, P.
;
Nelson, P.
;
Sharp, P.
;
Bindloss, C.
;
Dean, C.
;
Ravenscroft, E.
;
Fong, B.
;
Fietz, M.
;
Hopwood, J.
;
Meikle, P.
2001
Prediction of Sanfilippo phenotype severity from immunoquantification of heparan-N-sulfamidase in cultured fibroblasts from mucopolysaccharidosis type IIIA patients
Perkins, K.
;
Muller, V.
;
Weber, B.
;
Hopwood, J.
2000
Saposins A,B,C, and D in plasma of patients with lysosomal storage disorders
Chang, M.
;
Bindloss, C.
;
Grabowski, G.
;
Qi, X.
;
Winchester, B.
;
Hopwood, J.
;
Meikle, P.
Discover
Author
7
Harmatz, P.
7
Meikle, P.
6
Ketteridge, D.
5
Brooks, D.
5
Giugliani, R.
5
Guffon, N.
5
Miranda, C.
5
Swiedler, S.
5
Teles, E.
5
Wraith, J.
.
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Subject
22
Humans
20
Child
15
Adult
14
Child, Preschool
13
Male
12
Female
9
Infant
8
Mucopolysaccharidosis VI
8
Recombinant Proteins
7
N-Acetylgalactosamine-4-Sulfatase
.
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1996 - 1999