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TÍTULO: Design, baseline characteristics, and early findings of the MPS VI (mucopolysaccharidosis VI) Clinical Surveillance Program (CSP)  Full Text
AUTORES: Christian J Hendriksz; Roberto Giugliani; Paul Harmatz; Christina Lampe; Ana Maria Martins; Gregory M Pastores; Robert D Steiner; Elisa Leao Teles; Vassili Valayannopoulos;
PUBLICAÇÃO: 2013, FONTE: JOURNAL OF INHERITED METABOLIC DISEASE, VOLUME: 36, NÚMERO: 2
INDEXADO EM: Scopus WOS CrossRef
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TÍTULO: Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme (R)) therapy  Full Text
AUTORES: Braunlin, E; Rosenfeld, H; Kampmann, C; Johnson, J; Beck, M; Giugliani, R; Guffon, N; Ketteridge, D; Sa Miranda, CMS ; Scarpa, M; Schwartz, IV; Leao Teles, EL; Wraith, JE; Barrios, P; Dias da Silva, ED; Kurio, G; Richardson, M; Gildengorin, G; Hopwood, JJ; Imperiale, M; Schatz, A; Decker, C; Harmatz, P; ...Mais
PUBLICAÇÃO: 2013, FONTE: JOURNAL OF INHERITED METABOLIC DISEASE, VOLUME: 36, NÚMERO: 2
INDEXADO EM: Scopus WOS CrossRef
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TÍTULO: Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase  Full Text
AUTORES: Paul Harmatz; Zi Fan Yu; Roberto Giugliani; Ida Vanessa D Schwartz; Nathalie Guffon; Elisa Leao Teles; Clara C S Sa Miranda ; Edmond E Wraith; Michael Beck; Laila Arash; Maurizio Scarpa; David Ketteridge; John J Hopwood; Barbara Plecko; Robert Steiner; Chester B Whitley; Paige Kaplan; Stuart J Swiedler; Karen Hardy; Kenneth I Berger; Celeste Decker; ...Mais
PUBLICAÇÃO: 2010, FONTE: JOURNAL OF INHERITED METABOLIC DISEASE, VOLUME: 33, NÚMERO: 1
INDEXADO EM: Scopus WOS CrossRef
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TÍTULO: 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  Full Text
AUTORES: Paul Harmatz; Roberto Giugliani; Ida Vanessa D Schwartz; Nathalie Guffon; Elisa Leao Teles; Clara Sa S Miranda ; Edmond E Wraith; Michael Beck; Laila Arash; Maurizio Scarpa; David Ketteridge; John J Hopwood; Barbara Plecko; Robert Steiner; Chester B Whitley; Paige Kaplan; Zi Fan Yu; Stuart J Swiedler; Celeste Decker;
PUBLICAÇÃO: 2008, FONTE: MOLECULAR GENETICS AND METABOLISM, VOLUME: 94, NÚMERO: 4
INDEXADO EM: Scopus WOS CrossRef
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TÍTULO: Cardiac findings in MPS VI patients after enzyme replacement therapy  Full Text
AUTORES: Harmatz, P; Rosenfeld, H; Giugliani, R; Schwartz, I; Guffon, N; Sa S Miranda; Teles, EL; Wraith, JE; Beck, M; Scarpa, M; Mark Hudes; Sean Turbeville; Richard Tibbs; Swiedler, SJ; Decker, C;
PUBLICAÇÃO: 2007, FONTE: 3rd Annual World Symposium on Lysosomal Disease Networks in MOLECULAR GENETICS AND METABOLISM, VOLUME: 92, NÚMERO: 4
INDEXADO EM: WOS CrossRef
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TÍTULO: 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-label extension study  Full Text
AUTORES: Harmatz, P; Giugliani, R; Schwariz, I; Guffon, N; Teles, EL; Miranda, MCS ; Wraith, JE; Beck, M; Arash, L; Scarpa, M; Yu, ZF; Wittes, J; Berger, KI; Newman, MS; Lowe, AM; Kakkis, E; Swiedler, SJ;
PUBLICAÇÃO: 2006, FONTE: JOURNAL OF PEDIATRICS, VOLUME: 148, NÚMERO: 4
INDEXADO EM: Scopus WOS CrossRef
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