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Journal Abstract Search

297 related items for PubMed ID: 24764221

  • 1. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study.
    Giugliani R, Lampe C, Guffon N, Ketteridge D, Leão-Teles E, Wraith JE, Jones SA, Piscia-Nichols C, Lin P, Quartel A, Harmatz P.
    Am J Med Genet A; 2014 Aug; 164A(8):1953-64. PubMed ID: 24764221
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  • 2. Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program.
    Harmatz PR, Lampe C, Parini R, Sharma R, Teles EL, Johnson J, Sivam D, Sisic Z.
    J Inherit Metab Dis; 2019 May; 42(3):519-526. PubMed ID: 30834539
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  • 3. The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
    Harmatz P, Hendriksz CJ, Lampe C, McGill JJ, Parini R, Leão-Teles E, Valayannopoulos V, Cole TJ, Matousek R, Graham S, Guffon N, Quartel A, MPS VI Study Group.
    Mol Genet Metab; 2017 Sep; 122(1-2):107-112. PubMed ID: 28457718
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  • 4. 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
    [Abstract] [Full Text] [Related]

  • 5. Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI.
    Brunelli MJ, Atallah ÁN, da Silva EM.
    Cochrane Database Syst Rev; 2021 Sep 17; 9(9):CD009806. PubMed ID: 34533215
    [Abstract] [Full Text] [Related]

  • 6. 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 17; 115(6):e681-9. PubMed ID: 15930196
    [Abstract] [Full Text] [Related]

  • 7. 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 17; 133(1):100-108. PubMed ID: 33775523
    [Abstract] [Full Text] [Related]

  • 8. Enzyme replacement therapy initiated in adulthood: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program.
    Lampe C, Harmatz PR, Parini R, Sharma R, Teles EL, Johnson J, Sivam D, Sisic Z.
    Mol Genet Metab; 2019 Aug 17; 127(4):355-360. PubMed ID: 31324526
    [Abstract] [Full Text] [Related]

  • 9. Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI.
    Brunelli MJ, Atallah ÁN, da Silva EM.
    Cochrane Database Syst Rev; 2016 Mar 04; 3():CD009806. PubMed ID: 26943923
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  • 12. 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 04; 33(1):51-60. PubMed ID: 20140523
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  • 13. 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 04; 117(2):157-63. PubMed ID: 26776148
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  • 15. Enzyme replacement therapy with galsulfase for mucopolysaccharidosis VI: clinical facts and figures.
    Harmatz P.
    Turk J Pediatr; 2010 Feb 04; 52(5):443-9. PubMed ID: 21434527
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  • 17. Enzyme replacement therapy with galsulfase in 34 children younger than five years of age with MPS VI.
    Horovitz DD, Magalhães TS, Acosta A, Ribeiro EM, Giuliani LR, Palhares DB, Kim CA, de Paula AC, Kerstenestzy M, Pianovski MA, Costa MI, Santos FC, Martins AM, Aranda CS, Correa Neto J, Holanda GB, Cardoso L, da Silva CA, Bonatti RC, Ribeiro BF, Rodrigues Mdo C, Llerena JC.
    Mol Genet Metab; 2013 May 04; 109(1):62-9. PubMed ID: 23535281
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  • 18. Reversed papilledema in an MPS VI patient with galsulfase (Naglazyme) therapy.
    Koseoglu ST, Harmatz P, Turbeville S, Nicely H.
    Int Ophthalmol; 2009 Aug 04; 29(4):267-9. PubMed ID: 18418554
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  • 20. Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI): a single dose of galsulfase further reduces urine glycosaminoglycans after hematopoietic stem cell transplantation.
    Whitley CB, Utz JR.
    Mol Genet Metab; 2010 Dec 04; 101(4):346-8. PubMed ID: 20800524
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