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Items: 1 to 20 of 175

1.

Enzyme replacement therapy improves joint motion and outcome of the 12-min walk test in a mucopolysaccharidosis type VI patient previously treated with bone marrow transplantation.

Sohn YB, Park SW, Kim SH, Cho SY, Ji ST, Kwon EK, Han SJ, Oh SJ, Park YJ, Ko AR, Paik KH, Lee J, Lee DH, Jin DK.

Am J Med Genet A. 2012 May;158A(5):1158-63. doi: 10.1002/ajmg.a.35263. Epub 2012 Apr 11.

PMID:
22495825
4.

Long-term intra-articular administration of recombinant human N-acetylgalactosamine-4-sulfatase in feline mucopolysaccharidosis VI.

Auclair D, Hopwood JJ, Lemontt JF, Chen L, Byers S.

Mol Genet Metab. 2007 Aug;91(4):352-61. Epub 2007 Jun 1.

PMID:
17544310
5.

Enzyme replacement therapy for mucopolysaccharidosis VI from 8 weeks of age--a sibling control study.

McGill JJ, Inwood AC, Coman DJ, Lipke ML, de Lore D, Swiedler SJ, Hopwood JJ.

Clin Genet. 2010 May;77(5):492-8. doi: 10.1111/j.1399-0004.2009.01324.x. Epub 2009 Nov 23.

PMID:
19968667
6.
7.

Up to five years experience with 11 mucopolysaccharidosis type VI patients.

Brands MM, Oussoren E, Ruijter GJ, Vollebregt AA, van den Hout HM, Joosten KF, Hop WC, Plug I, van der Ploeg AT.

Mol Genet Metab. 2013 May;109(1):70-6. doi: 10.1016/j.ymgme.2013.02.013. Epub 2013 Mar 4.

PMID:
23523338
8.

Effect of enzyme replacement therapy on bone formation in a feline model of mucopolysaccharidosis type VI.

Byers S, Nuttall JD, Crawley AC, Hopwood JJ, Smith K, Fazzalari NL.

Bone. 1997 Nov;21(5):425-31.

PMID:
9356736
9.

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. doi: 10.1002/ajmg.a.36584. Epub 2014 Apr 24.

10.

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.

PMID:
16647419
11.

Enzyme replacement therapy for mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): experience in Hong Kong.

But WM, Wong MY, Chow JC, Chan WK, Ko WT, Wu SP, Wong ML, Miu TY, Tse WY, Hung WW, Fan TW, Shek CC.

Hong Kong Med J. 2011 Aug;17(4):317-24.

12.

Intrathecal administration of recombinant human N-acetylgalactosamine 4-sulfatase to a MPS VI patient with pachymeningitis cervicalis.

Muñoz-Rojas MV, Horovitz DD, Jardim LB, Raymundo M, Llerena JC Jr, de Magalhães Tde S, Vieira TA, Costa R, Kakkis E, Giugliani R.

Mol Genet Metab. 2010 Apr;99(4):346-50. doi: 10.1016/j.ymgme.2009.11.008. Epub 2009 Dec 5.

PMID:
20036175
13.

Reversed papilledema in an MPS VI patient with galsulfase (Naglazyme) therapy.

Koseoglu ST, Harmatz P, Turbeville S, Nicely H.

Int Ophthalmol. 2009 Aug;29(4):267-9. doi: 10.1007/s10792-008-9213-7. Epub 2008 Apr 17.

14.

Immune response to enzyme replacement therapy: 4-sulfatase epitope reactivity of plasma antibodies from MPS VI cats.

Turner CT, Hopwood JJ, Bond CS, Brooks DA.

Mol Genet Metab. 1999 Jul;67(3):194-205.

PMID:
10381327
15.

Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI.

Brunelli MJ, Atallah ÁN, da Silva EM.

Cochrane Database Syst Rev. 2016 Mar 4;3:CD009806. doi: 10.1002/14651858.CD009806.pub2. Review.

PMID:
26943923
16.

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.

PMID:
17672828
17.

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, 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, Yu ZF, Swiedler SJ, Decker C; MPS VI Study Group.

Mol Genet Metab. 2008 Aug;94(4):469-75. doi: 10.1016/j.ymgme.2008.04.001. Epub 2008 May 23.

PMID:
18502162
18.

Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI.

Furujo M, Kubo T, Kosuga M, Okuyama T.

Mol Genet Metab. 2011 Dec;104(4):597-602. doi: 10.1016/j.ymgme.2011.08.029. Epub 2011 Aug 28.

PMID:
21930407
19.

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 Jr, da Silva CA, Bonatti RC, Ribeiro BF, Rodrigues Mdo C, Llerena JC Jr.

Mol Genet Metab. 2013 May;109(1):62-9. doi: 10.1016/j.ymgme.2013.02.014. Epub 2013 Mar 5.

20.

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. doi: 10.1007/s10545-010-9212-5. Epub 2010 Oct 6.

PMID:
20924685

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