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Page 1
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.
Pediatrics. 2005 Jun;115(6):e681-9. doi: 10.1542/peds.2004-1023.
Pediatrics. 2005.
PMID: 15930196
Clinical Trial.
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.
Harmatz P, et al.
Mol Genet Metab. 2017 Sep;122(1-2):107-112. doi: 10.1016/j.ymgme.2017.03.008. Epub 2017 Mar 31.
Mol Genet Metab. 2017.
PMID: 28457718
Free article.
Clinical Trial.
Item in Clipboard
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.
Braunlin E, et al.
J Inherit Metab Dis. 2013 Mar;36(2):385-94. doi: 10.1007/s10545-012-9481-2. Epub 2012 Jun 5.
J Inherit Metab Dis. 2013.
PMID: 22669363
Free PMC article.
Item in Clipboard
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.
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.
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