1
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
2
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
3
TÍTULO: Mutational analysis of 105 mucopolysaccharidosis type VI patients  Full Text
AUTORES: Litsa Karageorgos; Doug A Brooks; Anthony Pollard; Elizabeth L Melville; Leanne K Hein; Peter R Clements; David Ketteridge; Stuart J Swiedler; Michael Beck; Roberto Giugliani; Paul Harmatz; James E Wraith; Nathalie Guffon; Elisa Leao Teles; Clara Sa S Miranda ; John J Hopwood;
PUBLICAÇÃO: 2007, FONTE: HUMAN MUTATION, VOLUME: 28, NÚMERO: 9
INDEXADO EM: Scopus WOS CrossRef
4
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