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Items: 1 to 20 of 304

1.

Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.

Tolar J, Grewal SS, Bjoraker KJ, Whitley CB, Shapiro EG, Charnas L, Orchard PJ.

Bone Marrow Transplant. 2008 Mar;41(6):531-5. Epub 2007 Nov 26.

PMID:
18037941
2.

Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome.

Cox-Brinkman J, Boelens JJ, Wraith JE, O'meara A, Veys P, Wijburg FA, Wulffraat N, Wynn RF.

Bone Marrow Transplant. 2006 Jul;38(1):17-21. Epub 2006 May 22.

PMID:
16715104
3.

Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome.

Grewal SS, Wynn R, Abdenur JE, Burton BK, Gharib M, Haase C, Hayashi RJ, Shenoy S, Sillence D, Tiller GE, Dudek ME, van Royen-Kerkhof A, Wraith JE, Woodard P, Young GA, Wulffraat N, Whitley CB, Peters C.

Genet Med. 2005 Feb;7(2):143-6.

PMID:
15714083
4.

Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation.

Polgreen LE, Tolar J, Plog M, Himes JH, Orchard PJ, Whitley CB, Miller BS, Petryk A.

Bone Marrow Transplant. 2008 Jun;41(12):1005-11. doi: 10.1038/bmt.2008.20. Epub 2008 Feb 18.

PMID:
18278070
5.

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.

6.

Residual α-L-iduronidase activity in fibroblasts of mild to severe Mucopolysaccharidosis type I patients.

Oussoren E, Keulemans J, van Diggelen OP, Oemardien LF, Timmermans RG, van der Ploeg AT, Ruijter GJ.

Mol Genet Metab. 2013 Aug;109(4):377-81. doi: 10.1016/j.ymgme.2013.05.016. Epub 2013 Jun 4.

PMID:
23786846
7.

Mucopolysaccharidosis type I.

Wraith JE, Jones S.

Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:102-6. Review.

PMID:
25345091
8.

Analysis of factors affecting development of carpal tunnel syndrome in patients with Hurler syndrome after hematopoietic cell transplantation.

Khanna G, Van Heest AE, Agel J, Bjoraker K, Grewal S, Abel S, Krivit W, Peters C, Orchard PJ.

Bone Marrow Transplant. 2007 Mar;39(6):331-4. Epub 2007 Feb 5.

PMID:
17277793
9.

Combined enzyme replacement and haematopoietic stem cell transplantation in Hurler syndrome.

Bijarnia S, Shaw P, Vimpani A, Smith R, Pacey V, O'Grady H, Christodoulou J, Sillence D.

J Paediatr Child Health. 2009 Jul-Aug;45(7-8):469-72. doi: 10.1111/j.1440-1754.2009.01537.x.

PMID:
19712183
10.

Dried blood spot analysis: an easy and reliable tool to monitor the biochemical effect of hematopoietic stem cell transplantation in hurler syndrome patients.

Aldenhoven M, de Koning TJ, Verheijen FW, Prinsen BH, Wijburg FA, van der Ploeg AT, de Sain-van der Velden MG, Boelens J.

Biol Blood Marrow Transplant. 2010 May;16(5):701-4. doi: 10.1016/j.bbmt.2010.01.006. Epub 2010 Jan 21.

11.

Successful treatment of severe heart failure in an infant with Hurler syndrome.

Hirth A, Berg A, Greve G.

J Inherit Metab Dis. 2007 Oct;30(5):820. Epub 2007 Sep 4.

PMID:
17768668
12.

Outcome in six children with mucopolysaccharidosis type IH, Hurler syndrome, after haematopoietic stem cell transplantation (HSCT).

Malm G, Gustafsson B, Berglund G, Lindström M, Naess K, Borgström B, von Döbeln U, Ringdén O.

Acta Paediatr. 2008 Aug;97(8):1108-12. doi: 10.1111/j.1651-2227.2008.00811.x. Epub 2008 Apr 29.

PMID:
18452566
13.
14.

Hematopoietic stem cell transplantation effects on spinal cord compression in Hurler.

Ferrara G, Maximova N, Zennaro F, Gregori M, Tamaro P.

Pediatr Transplant. 2014 May;18(3):E96-9. doi: 10.1111/petr.12231. Epub 2014 Feb 1.

PMID:
24483599
16.

Developmental outcome in five children with Hurler syndrome after stem cell transplantation: a pilot study.

Lücke T, Das AM, Hartmann H, Sykora KW, Donnerstag F, Schmid-Ott G, Grigull L.

Dev Med Child Neurol. 2007 Sep;49(9):693-6.

17.

Ultrastructural analysis of dermal fibroblasts in mucopolysaccharidosis type I: Effects of enzyme replacement therapy and hematopoietic cell transplantation.

Cox-Brinkman J, van den Bergh Weerman MA, Wijburg FA, Aerts JM, Florquin S, van der Lee JH, Hollak CE.

Ultrastruct Pathol. 2010 May;34(3):126-32. doi: 10.3109/01913121003648485.

PMID:
20455661
18.

Four novel mutations underlying mild or intermediate forms of alpha-L-iduronidase deficiency (MPS IS and MPS IH/S).

Tieu PT, Bach G, Matynia A, Hwang M, Neufeld EF.

Hum Mutat. 1995;6(1):55-9.

PMID:
7550232
19.

Life-threatening pulmonary hemorrhages post bone marrow transplantation in Hurler syndrome. Report of three cases and review of the literature.

Gassas A, Sung L, Doyle JJ, Clarke JT, Saunders EF.

Bone Marrow Transplant. 2003 Jul;32(2):213-5. Review.

PMID:
12838287
20.

Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients.

Wynn RF, Mercer J, Page J, Carr TF, Jones S, Wraith JE.

J Pediatr. 2009 Jan;154(1):135-9. doi: 10.1016/j.jpeds.2008.07.004.

PMID:
19187736

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