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Items: 1 to 20 of 43

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.

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.

3.

Mucopolysaccharidosis type I.

Wraith JE, Jones S.

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

PMID:
25345091
4.

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
5.

Alpha-L-iduronidase and enzyme replacement therapy for mucopolysaccharidosis I.

Brooks DA.

Expert Opin Biol Ther. 2002 Dec;2(8):967-76. Review.

PMID:
12517274
6.

Laronidase.

[No authors listed]

BioDrugs. 2002;16(4):316-8. Review.

PMID:
12196045
7.

Therapy for the mucopolysaccharidoses.

Valayannopoulos V, Wijburg FA.

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

PMID:
22210671
8.

Enzyme replacement therapy in mucopolysaccharidosis type I: progress and emerging difficulties.

Wraith JE.

J Inherit Metab Dis. 2001 Apr;24(2):245-50. Review.

PMID:
11405343
9.

Enzyme replacement therapy for the management of the mucopolysaccharidoses.

Wraith JE.

Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S63-5. Review.

PMID:
20040314
10.

Laronidase treatment of mucopolysaccharidosis I.

Wraith EJ, Hopwood JJ, Fuller M, Meikle PJ, Brooks DA.

BioDrugs. 2005;19(1):1-7. Review.

PMID:
15691212
11.

Enzyme replacement therapy in mucopolysaccharidosis type I.

Miebach E.

Acta Paediatr Suppl. 2005 Mar;94(447):58-60; discussion 57. Review.

PMID:
15895714
12.
13.

Transplant outcomes in mucopolysaccharidoses.

Prasad VK, Kurtzberg J.

Semin Hematol. 2010 Jan;47(1):59-69. doi: 10.1053/j.seminhematol.2009.10.008. Review.

PMID:
20109613
14.

Unrelated donor and HLA-identical sibling haematopoietic stem cell transplantation cure chronic granulomatous disease with good long-term outcome and growth.

Soncini E, Slatter MA, Jones LB, Hughes S, Hodges S, Flood TJ, Barge D, Spickett GP, Jackson GH, Collin MP, Abinun M, Cant AJ, Gennery AR.

Br J Haematol. 2009 Apr;145(1):73-83. doi: 10.1111/j.1365-2141.2009.07614.x. Epub 2009 Feb 16. Review.

PMID:
19222467
15.
16.

Laronidase (Aldurazyme): enzyme replacement therapy for mucopolysaccharidosis type I.

Pastores GM.

Expert Opin Biol Ther. 2008 Jul;8(7):1003-9. doi: 10.1517/14712598.8.7.1003 . Review.

PMID:
18549329
17.

Neuropsychological development in children with Hurler syndrome following hematopoietic stem cell transplantation.

Peters C, Shapiro EG, Krivit W.

Pediatr Transplant. 1998 Nov;2(4):250-3. Review. No abstract available.

PMID:
10084724
18.

Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.

Tomatsu S, Sawamoto K, Alméciga-Díaz CJ, Shimada T, Bober MB, Chinen Y, Yabe H, Montaño AM, Giugliani R, Kubaski F, Yasuda E, Rodríguez-López A, Espejo-Mojica AJ, Sánchez OF, Mason RW, Barrera LA, Mackenzie WG, Orii T.

Drug Des Devel Ther. 2015 Apr 1;9:1937-53. doi: 10.2147/DDDT.S68562. eCollection 2015. Review.

19.

Trends in haematopoietic cell transplantation for inborn errors of metabolism.

Boelens JJ.

J Inherit Metab Dis. 2006 Apr-Jun;29(2-3):413-20. Review.

PMID:
16763911
20.

Chronic lung disease after hematopoietic stem cell transplantation.

Afessa B, Peters SG.

Clin Chest Med. 2005 Dec;26(4):571-86, vi. Review.

PMID:
16263397

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