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Journal Abstract Search

179 related items for PubMed ID: 20140523

  • 1. Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.
    Harmatz P, Yu ZF, Giugliani R, Schwartz IV, Guffon N, Teles EL, Miranda MC, Wraith JE, Beck M, Arash L, Scarpa M, Ketteridge D, Hopwood JJ, Plecko B, Steiner R, Whitley CB, Kaplan P, Swiedler SJ, Hardy K, Berger KI, Decker C.
    J Inherit Metab Dis; 2010 Feb; 33(1):51-60. PubMed ID: 20140523
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  • 2. 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 P, Ketteridge D, Giugliani R, Guffon N, Teles EL, Miranda MC, Yu ZF, Swiedler SJ, Hopwood JJ, MPS VI Study Group.
    Pediatrics; 2005 Jun; 115(6):e681-9. PubMed ID: 15930196
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  • 4. 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, D Schwartz IV, Guffon N, Teles EL, Miranda MCS, Wraith JE, Beck M, Arash L, Scarpa M, Ketteridge D, Hopwood JJ, Plecko B, Steiner R, Whitley CB, Kaplan P, Yu ZF, Swiedler SJ, Decker C, MPS VI Study Group.
    Mol Genet Metab; 2008 Aug; 94(4):469-475. PubMed ID: 18502162
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  • 5. 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.
    Harmatz P, Giugliani R, Schwartz I, Guffon N, Teles EL, Miranda MC, Wraith JE, Beck M, Arash L, Scarpa M, Yu ZF, Wittes J, Berger KI, Newman MS, Lowe AM, Kakkis E, Swiedler SJ, MPS VI Phase 3 Study Group.
    J Pediatr; 2006 Apr; 148(4):533-539. PubMed ID: 16647419
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  • 6. 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, Sá Miranda CM, Scarpa M, Schwartz IV, Leão Teles E, Wraith JE, Barrios P, Dias da Silva E, Kurio G, Richardson M, Gildengorin G, Hopwood JJ, Imperiale M, Schatz A, Decker C, Harmatz P, MPS VI Study Group.
    J Inherit Metab Dis; 2013 Mar; 36(2):385-94. PubMed ID: 22669363
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  • 7. Intrathecal recombinant human 4-sulfatase reduces accumulation of glycosaminoglycans in dura of mucopolysaccharidosis VI cats.
    Auclair D, Finnie J, Walkley SU, White J, Nielsen T, Fuller M, Cheng A, O'Neill CA, Hopwood JJ.
    Pediatr Res; 2012 Jan; 71(1):39-45. PubMed ID: 22289849
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  • 8. Galsulfase: arylsulfatase B, BM 102, recombinant human arylsulfatase B, recombinant human N-acetylgalactosamine-4-sulfatase, rhASB.
    Drugs R D; 2005 Jan; 6(5):312-5. PubMed ID: 16128602
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  • 10. Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy.
    Garcia P, Phillips D, Johnson J, Martin K, Randolph LM, Rosenfeld H, Harmatz P.
    Mol Genet Metab; 2021 May; 133(1):100-108. PubMed ID: 33775523
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  • 11. Enzyme replacement therapy for mucopolysaccharidosis VI--experience in Taiwan.
    Lin HY, Chen MR, Chuang CK, Chen CP, Lin DS, Chien YH, Ke YY, Tsai FJ, Pan HP, Lin SJ, Hwu WL, Niu DM, Lee NC, Lin SP.
    J Inherit Metab Dis; 2010 Dec; 33 Suppl 3():S421-7. PubMed ID: 20924685
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  • 12. Pharmacodynamics, pharmacokinetics and biodistribution of recombinant human N-acetylgalactosamine 4-sulfatase after 6months of therapy in cats using different IV infusion durations.
    Ruane T, Haskins M, Cheng A, Wang P, Aguirre G, Knox VW, Qi Y, Tompkins T, O'Neill CA.
    Mol Genet Metab; 2016 Feb; 117(2):157-63. PubMed ID: 26776148
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  • 14. Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study.
    Horovitz DDG, Leão EKEA, Ribeiro EM, Martins AM, Barth AL, Neri JICF, Kerstenetzky M, Siqueira ACM, Ribeiro BFR, Kim CA, Santos FC, Franco JFS, Lichtvan LCL, Giuliani LR, Rodrigues MDCS, Bonatti RCF, Teixeira TB, Gonçalves A, Lourenço CM, Pereira ASS, Acosta AX.
    Mol Genet Metab; 2021 May; 133(1):94-99. PubMed ID: 33678523
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  • 19. Molecular markers for the follow-up of enzyme-replacement therapy in mucopolysaccharidosis type VI disease.
    Di Natale P, Villani GR, Parini R, Scarpa M, Parenti G, Pontarelli G, Grosso M, Sersale G, Tomanin R, Sibilio M, Barone R, Fiumara A.
    Biotechnol Appl Biochem; 2008 Mar; 49(Pt 3):219-23. PubMed ID: 17672828
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