Enzyme replacement therapy for mucopolysaccharidosis VI from 8 weeks of age-a sibling control study
McGill, JJ, Inwood, AC, Coman, DJ, Lipke, ML, De Lore, D, Swiedler, SJ, Hopwood, JJ
Published in Clinical genetics (01.05.2010)
Published in Clinical genetics (01.05.2010)
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Cord-Blood Transplants from Unrelated Donors in Patients with Hurler's Syndrome
Staba, Susan L, Escolar, Maria L, Poe, Michele, Kim, Young, Martin, Paul L, Szabolcs, Paul, Allison-Thacker, June, Wood, Susan, Wenger, David A, Rubinstein, Pablo, Hopwood, John J, Krivit, William, Kurtzberg, Joanne
Published in The New England journal of medicine (06.05.2004)
Published in The New England journal of medicine (06.05.2004)
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Prevalence of Lysosomal Storage Disorders
Meikle, Peter J, Hopwood, John J, Clague, Alan E, Carey, William F
Published in JAMA : the journal of the American Medical Association (20.01.1999)
Published in JAMA : the journal of the American Medical Association (20.01.1999)
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Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Harmatz, Paul, Whitley, Chester B, Waber, Lewis, Pais, Ray, Steiner, Robert, Plecko, Barbara, Kaplan, Paige, Simon, Julie, Butensky, Ellen, Hopwood, John J
Published in The Journal of pediatrics (01.05.2004)
Published in The Journal of pediatrics (01.05.2004)
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Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase
Harmatz, Paul, Ketteridge, David, Giugliani, Roberto, Guffon, Natalie, Teles, Elisa Leão, Miranda, M Clara Sá, Yu, Zi-Fan, Swiedler, Stuart J, Hopwood, John J
Published in Pediatrics (Evanston) (01.06.2005)
Published in Pediatrics (Evanston) (01.06.2005)
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Replacing acid α-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers
Raben, Nina, Fukuda, Tokiko, Gilbert, Abigail L., de Jong, Deborah, Thurberg, Beth L., Mattaliano, Robert J., Meikle, Peter, Hopwood, John J., Nagashima, Kunio, Nagaraju, Kanneboyina, Plotz, Paul H.
Published in Molecular therapy (01.01.2005)
Published in Molecular therapy (01.01.2005)
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Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Swiedler, Stuart J., Beck, Michael, Bajbouj, Manal, Giugliani, Roberto, Schwartz, Ida, Harmatz, Paul, Wraith, James E., Roberts, Jane, Ketteridge, David, Hopwood, John J., Guffon, Nathalie, Sá Miranda, M. Clara, Teles, Elisa Leão, Berger, Kenneth I., Piscia-Nichols, Cheri
Published in American journal of medical genetics. Part A (15.04.2005)
Published in American journal of medical genetics. Part A (15.04.2005)
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Newborn screening for lysosomal storage disorders: clinical evaluation of a two-tier strategy
Meikle, Peter J, Ranieri, Enzo, Simonsen, Henrik, Rozaklis, Tina, Ramsay, Steve L, Whitfield, Phillip D, Fuller, Maria, Christensen, Ernst, Skovby, Flemming, Hopwood, John J
Published in Pediatrics (Evanston) (01.10.2004)
Published in Pediatrics (Evanston) (01.10.2004)
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Journal Article
Gentamicin-mediated suppression of Hurler syndrome stop mutations restores a low level of α-L-iduronidase activity and reduces lysosomal glycosaminoglycan accumulation
KEELING, Kim M, BROOKS, Doug A, HOPWOOD, John J, PEINING LI, THOMPSON, Jerry N, BEDWELL, David M
Published in Human molecular genetics (01.02.2001)
Published in Human molecular genetics (01.02.2001)
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Journal Article
Usefulness of bone marrow transplantation in the Hurler syndrome
Braunlin, Elizabeth A, Stauffer, Nanci R, Peters, Charles H, Bass, John L, Berry, James M, Hopwood, John J, Krivit, William
Published in The American journal of cardiology (01.10.2003)
Published in The American journal of cardiology (01.10.2003)
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Intracerebral injection of sulfamidase delays neuropathology in murine MPS-IIIA
Savas, Peter S, Hemsley, Kim M, Hopwood, John J
Published in Molecular genetics and metabolism (01.08.2004)
Published in Molecular genetics and metabolism (01.08.2004)
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Journal Article
Galsulfase
Hopwood, John J, Bate, Guy, Kirkpatrick, Peter
Published in Nature reviews. Drug discovery (01.02.2006)
Published in Nature reviews. Drug discovery (01.02.2006)
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