%0 Journal Article %J Journal of Inherited Metabolic Disease %D 2013 %T Enzyme replacement therapy for mucopolysaccharidosis VI: Long-term cardiac effects of galsulfase (Naglazyme®) therapy %A Braunlin, E. %A Rosenfeld, H. %A Kampmann, C. %A Johnson, J. %A Beck, M. %A Giugliani, R. %A Guffon, N. %A Ketteridge, D. %A SáMiranda, C. M. %A Scarpa, M. %A Schwartz, I. V. %A Leão Teles, E. %A Wraith, J.E. %A Barrios, P. %A Dias da Silva, E. %A Kurio, G. %A Richardson, M. %A Gildengorin, G. %A Hopwood, J.J. %A Imperiale, M. %A Schatz, A. %A Decker, C. %A Harmatz, P. %B Journal of Inherited Metabolic Disease %V 36 %P 385 - 394 %8 2013 %G eng %U http://www.scopus.com/inward/record.url?eid=2-s2.0-84879684477&partnerID=40&md5=809f269d1e1f742fd6dc536bbf98b56b %N 2 %0 Generic %D 2012 %T Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme ®) therapy %A Braunlin, E. %A Rosenfeld, H. %A Kampmann, C. %A Johnson, J. %A Beck, M. %A Giugliani, R. %A Guffon, N. %A Ketteridge, D. %A SáMiranda, C. M. %A Scarpa, M. %A Schwartz, I. V. %A Leão Teles, E. %A Wraith, J.E. %A Barrios, P. %A Dias da Silva, E. %A Kurio, G. %A Richardson, M. %A Gildengorin, G. %A Hopwood, J.J. %A Imperiale, M. %A Schatz, A. %A Decker, C. %A Harmatz, P. %B Journal of Inherited Metabolic Disease %P 1 - 10 %8 2012/// %G eng %U http://www.scopus.com/inward/record.url?eid=2-s2.0-84861722683&partnerID=40&md5=cada11c663bff78563ca271d528303bb %0 Journal Article %J Journal of Inherited Metabolic Disease %D 2010 %T Enzyme replacement therapy for mucopolysaccharidosis VI: Evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase %A Harmatz, P. %A Yu, Z.-F. %A Giugliani, R. %A Schwartz, I.V.D. %A Guffon, N. %A Teles, E.L. %A Miranda, M.C.S. %A Wraith, J.E. %A Beck, M. %A Arash, L. %A Scarpa, M. %A Ketteridge, D. %A Hopwood, J.J. %A Plecko, B. %A Steiner, R. %A Whitley, C.B. %A Kaplan, P. %A Swiedler, S.J. %A Hardy, K. %A Berger, K.I. %A Decker, C. %B Journal of Inherited Metabolic Disease %V 33 %P 51 - 60 %8 2010/// %G eng %U http://www.scopus.com/inward/record.url?eid=2-s2.0-77649237484&partnerID=40&md5=b0a62e125234c3e1d9c52bde100b1f47 %N 1 %0 Journal Article %J Journal of Pediatric Rehabilitation Medicine %D 2010 %T Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase %A Decker, C. %A Yu, Z.-F. %A Giugliani, R. %A Schwartz, I.V.D. %A Guffon, N. %A Teles, E.L. %A Miranda, M.C.S. %A Wraith, J.E. %A Beck, M. %A Arash, L. %A Scarpa, M. %A Ketteridge, D. %A Hopwood, J.J. %A Plecko, B. %A Steiner, R. %A Whitley, C.B. %A Kaplan, P. %A Swiedler, S.J. %A Conrad, S. %A Harmatz, P. %B Journal of Pediatric Rehabilitation Medicine %V 3 %P 89 - 100 %8 2010/// %G eng %U http://www.scopus.com/inward/record.url?eid=2-s2.0-77954629021&partnerID=40&md5=9d8b9a8582dbf72c9ab58a33205e09a7 %N 2 %0 Journal Article %J Molecular Genetics and Metabolism %D 2008 %T 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 %A Harmatz, P. %A Giugliani, R. %A D. Schwartz, I.V. %A Guffon, N. %A Teles, E.L. %A Miranda, M.C.S. %A Wraith, J.E. %A Beck, M. %A Arash, L. %A Scarpa, M. %A Ketteridge, D. %A Hopwood, J.J. %A Plecko, B. %A Steiner, R. %A Whitley, C.B. %A Kaplan, P. %A Yu, Z.-F. %A Swiedler, S.J. %A Decker, C. %B Molecular Genetics and Metabolism %V 94 %P 469 - 475 %8 2008/// %G eng %U http://www.scopus.com/inward/record.url?eid=2-s2.0-46949093352&partnerID=40&md5=3cf7db34b2fa5e51f33003e05d528b75 %N 4 %0 Journal Article %J Human Mutation %D 2007 %T Mutational analysis of 105 mucopolysaccharidosis type VI patients %A Karageorgos, L. %A Brooks, D.A. %A Pollard, A. %A Melville, E.L. %A Hein, L.K. %A Clements, P.R. %A Ketteridge, D. %A Swiedler, S.J. %A Beck, M. %A Giugliani, R. %A Harmatz, P. %A Wraith, J.E. %A Guffon, N. %A Teles, E.L. %A Sá Miranda, M.C. %A Hopwood, J.J. %B Human Mutation %V 28 %P 897 - 903 %8 2007/// %G eng %U http://www.scopus.com/inward/record.url?eid=2-s2.0-34547673433&partnerID=40&md5=a23ac7304a5def19685c11fa229c1879 %N 9 %0 Journal Article %J Journal of Pediatrics %D 2006 %T Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-l %A Harmatz, P. %A Giugliani, R. %A Schwartz, I. %A Guffon, N. %A Teles, E.L. %A Miranda, M.C.S. %A Wraith, J.E. %A Beck, M. %A Arash, L. %A Scarpa, M. %A Yu, Z.-F. %A Wittes, J. %A Berger, K.I. %A Newman, M.S. %A Lowe, A.M. %A Kakkis, E. %A Swiedler, S.J. %B Journal of Pediatrics %V 148 %P 533 - 533.e %8 2006/// %G eng %U http://www.scopus.com/inward/record.url?eid=2-s2.0-33744978567&partnerID=40&md5=0620094eea7917a76536626611bb8a8c %N 4 %0 Journal Article %J Pediatrics %D 2005 %T 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-acetylga %A Harmatz, P. %A Ketteridge, D. %A Giugliani, R. %A Guffon, N. %A Teles, E.L. %A Miranda, M.C.S. %A Yu, Z.-F. %A Swiedler, S.J. %A Hopwood, J.J. %B Pediatrics %V 115 %P e681 - e689 %8 2005/// %G eng %U http://www.scopus.com/inward/record.url?eid=2-s2.0-27744493202&partnerID=40&md5=5cd4badc420144129a7b5092723f0d06 %N 6 %0 Journal Article %J American Journal of Medical Genetics %D 2005 %T 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) %A Swiedler, S.J. %A Beck, M. %A Bajbouj, M. %A Giugliani, R. %A Schwartz, I. %A Harmatz, P. %A Wraith, J.E. %A Roberts, J. %A Ketteridge, D. %A Hopwood, J.J. %A Guffon, N. %A Sá Miranda, M.C. %A Teles, E.L. %A Berger, K.I. %A Piscia-Nichols, C. %B American Journal of Medical Genetics %V 134 A %P 144 - 150 %8 2005/// %G eng %U http://www.scopus.com/inward/record.url?eid=2-s2.0-20144386995&partnerID=40&md5=9c8cfc3e51a0cff4dbcd8a9b70a70aad %N 2