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31 results

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Page 1
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).
Swiedler SJ, Beck M, Bajbouj M, Giugliani R, Schwartz I, Harmatz P, Wraith JE, Roberts J, Ketteridge D, Hopwood JJ, Guffon N, Sá Miranda MC, Teles EL, Berger KI, Piscia-Nichols C. Swiedler SJ, et al. Am J Med Genet A. 2005 Apr 15;134A(2):144-50. doi: 10.1002/ajmg.a.30579. Am J Med Genet A. 2005. PMID: 15690405
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. Harmatz P, et al. Among authors: swiedler sj. Mol Genet Metab. 2008 Aug;94(4):469-475. doi: 10.1016/j.ymgme.2008.04.001. Epub 2008 May 23. Mol Genet Metab. 2008. PMID: 18502162 Clinical Trial.
Pharmacokinetic profile of recombinant human N-acetylgalactosamine 4-sulphatase enzyme replacement therapy in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): a phase I/II study.
Harmatz P, Kramer WG, Hopwood JJ, Simon J, Butensky E, Swiedler SJ; Mucopolysaccharidosis VI Study Group. Harmatz P, et al. Among authors: swiedler sj. Acta Paediatr Suppl. 2005 Mar;94(447):61-8; discussion 57. doi: 10.1111/j.1651-2227.2005.tb02115.x. Acta Paediatr Suppl. 2005. PMID: 15895715 Clinical Trial.
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. Harmatz P, et al. Among authors: swiedler sj. Pediatrics. 2005 Jun;115(6):e681-9. doi: 10.1542/peds.2004-1023. Pediatrics. 2005. PMID: 15930196 Clinical Trial.
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. Harmatz P, et al. Among authors: swiedler sj. J Pediatr. 2006 Apr;148(4):533-539. doi: 10.1016/j.jpeds.2005.12.014. J Pediatr. 2006. PMID: 16647419 Clinical Trial.
Mutational analysis of 105 mucopolysaccharidosis type VI patients.
Karageorgos L, Brooks DA, Pollard A, Melville EL, Hein LK, Clements PR, Ketteridge D, Swiedler SJ, Beck M, Giugliani R, Harmatz P, Wraith JE, Guffon N, Leão Teles E, Sá Miranda MC, Hopwood JJ. Karageorgos L, et al. Among authors: swiedler sj. Hum Mutat. 2007 Sep;28(9):897-903. doi: 10.1002/humu.20534. Hum Mutat. 2007. PMID: 17458871
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. Harmatz P, et al. Among authors: swiedler sj. J Inherit Metab Dis. 2010 Feb;33(1):51-60. doi: 10.1007/s10545-009-9007-8. Epub 2010 Feb 6. J Inherit Metab Dis. 2010. PMID: 20140523 Free PMC article. Clinical Trial.
Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.
Decker C, 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, Conrad S, Harmatz P. Decker C, et al. Among authors: swiedler sj. J Pediatr Rehabil Med. 2010;3(2):89-100. doi: 10.3233/PRM-2010-0113. J Pediatr Rehabil Med. 2010. PMID: 20634905 Free PMC article.
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).
Wraith JE, Clarke LA, Beck M, Kolodny EH, Pastores GM, Muenzer J, Rapoport DM, Berger KI, Swiedler SJ, Kakkis ED, Braakman T, Chadbourne E, Walton-Bowen K, Cox GF. Wraith JE, et al. Among authors: swiedler sj. J Pediatr. 2004 May;144(5):581-8. doi: 10.1016/j.jpeds.2004.01.046. J Pediatr. 2004. PMID: 15126990 Clinical Trial.
31 results