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Items: 1 to 20 of 118

1.

Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI.

Turbeville S, Nicely H, Rizzo JD, Pedersen TL, Orchard PJ, Horwitz ME, Horwitz EM, Veys P, Bonfim C, Al-Seraihy A.

Mol Genet Metab. 2011 Feb;102(2):111-5. doi: 10.1016/j.ymgme.2010.09.010. Epub 2010 Oct 25.

2.
3.

Haploidentical stem cell transplantation in two children with mucopolysaccharidosis VI: clinical and biochemical outcome.

Jester S, Larsson J, Eklund EA, Papadopoulou D, Månsson JE, Békássy AN, Turkiewicz D, Toporski J, Øra I.

Orphanet J Rare Dis. 2013 Sep 5;8:134. doi: 10.1186/1750-1172-8-134.

4.

Therapy for mucopolysaccharidosis VI: (Maroteaux-Lamy syndrome) present status and prospects.

Giugliani R, Herber S, Lapagesse L, de Pinto C, Baldo G.

Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:152-8. Review.

PMID:
25345097
5.

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.

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7.

Combined Enzyme Replacement Therapy and Hematopoietic Stem Cell Transplantation in Mucopolysacharidosis Type VI.

Sillence D, Waters K, Donaldson S, Shaw PJ, Ellaway C.

JIMD Rep. 2012;2:103-6. doi: 10.1007/8904_2011_56. Epub 2011 Sep 6.

8.

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
9.

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.

11.

Abnormal granulation of blood granulocytes in mucopolysaccharidosis VI-a case report.

Krishnagiri C, Ajanahalli RR, Kashyap S, Anegundi R, Boranaik L.

Ann Diagn Pathol. 2013 Feb;17(1):137-9. doi: 10.1016/j.anndiagpath.2011.07.009. Epub 2011 Nov 4.

PMID:
22056033
12.

Mucopolysaccharidosis VI.

Valayannopoulos V, Nicely H, Harmatz P, Turbeville S.

Orphanet J Rare Dis. 2010 Apr 12;5:5. doi: 10.1186/1750-1172-5-5. Review.

13.

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
15.

Therapy for the mucopolysaccharidoses.

Valayannopoulos V, Wijburg FA.

Rheumatology (Oxford). 2011 Dec;50 Suppl 5:v49-59. doi: 10.1093/rheumatology/ker396. Review.

17.

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
18.

Umbilical cord blood transplantation for Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI).

Lee V, Li CK, Shing MM, Chik KW, Lam CW, Tsang KS, Pong H, Huen KF, Yuen PM.

Bone Marrow Transplant. 2000 Aug;26(4):455-8.

19.

Epileptic seizures in patients following allogeneic hematopoietic stem cell transplantation: a retrospective analysis of incidence, risk factors, and survival rates.

Zhang XH, Xu LP, Liu DH, Chen H, Han W, Chen YH, Wang FR, Wang JZ, Wang Y, Zhao T, Chen Y, Fu HX, Liu KY, Huang XJ.

Clin Transplant. 2013 Jan-Feb;27(1):80-9. doi: 10.1111/ctr.12000. Epub 2012 Sep 2.

PMID:
22938074
20.

Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure.

de Ru MH, Boelens JJ, Das AM, Jones SA, van der Lee JH, Mahlaoui N, Mengel E, Offringa M, O'Meara A, Parini R, Rovelli A, Sykora KW, Valayannopoulos V, Vellodi A, Wynn RF, Wijburg FA.

Orphanet J Rare Dis. 2011 Aug 10;6:55. doi: 10.1186/1750-1172-6-55.

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