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Items: 1 to 20 of 102

1.

Long-term adaptive functioning outcomes of children with inherited metabolic and genetic diseases treated with hematopoietic stem cell transplantation in a single large pediatric center: parents' perspective.

Gassas A, Raiman J, White L, Schechter T, Clarke J, Doyle J.

J Pediatr Hematol Oncol. 2011 Apr;33(3):216-20. doi: 10.1097/MPH.0b013e3182050945.

PMID:
21336168
2.
3.

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines.

Peters C, Steward CG; National Marrow Donor Program; International Bone Marrow Transplant Registry; Working Party on Inborn Errors, European Bone Marrow Transplant Group.

Bone Marrow Transplant. 2003 Feb;31(4):229-39. Review.

PMID:
12621457
4.

Bone marrow transplantation for genetic and metabolic disorders.

Schaison G, Bordigoni P, Leverger G.

Nouv Rev Fr Hematol. 1989;31(2):119-23. Review.

PMID:
2505227
5.
6.

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
7.

Outcome of second hematopoietic cell transplantation in Hurler syndrome.

Grewal SS, Krivit W, Defor TE, Shapiro EG, Orchard PJ, Abel SL, Lockman LA, Ziegler RS, Dusenbery KE, Peters C.

Bone Marrow Transplant. 2002 Mar;29(6):491-6.

8.

Unrelated donor marrow transplantation for congenital immunodeficiency and metabolic disease: an update of the experience of the Japan Marrow Donor Program.

Sakata N, Kawa K, Kato K, Yabe H, Yabe M, Nagasawa M, Mugishima H, Kigasawa H, Tsuchida M, Akiyama Y, Morisima Y, Kodera Y, Kato S.

Int J Hematol. 2004 Aug;80(2):174-82.

PMID:
15481448
9.
10.

Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report.

Driessen GJ, Gerritsen EJ, Fischer A, Fasth A, Hop WC, Veys P, Porta F, Cant A, Steward CG, Vossen JM, Uckan D, Friedrich W.

Bone Marrow Transplant. 2003 Oct;32(7):657-63.

PMID:
13130312
11.

Second hematopoietic stem cell transplantation in pediatric patients: overall survival and long-term follow-up.

Shah AJ, Kapoor N, Weinberg KI, Crooks GM, Kohn DB, Lenarsky C, Kaufman F, Epport K, Wilson K, Parkman R.

Biol Blood Marrow Transplant. 2002;8(4):221-8.

PMID:
12014811
12.

Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases.

Krivit W.

Springer Semin Immunopathol. 2004 Nov;26(1-2):119-32. Epub 2004 Sep 25. Review.

PMID:
15452666
13.

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
14.

Haematopoietic transplant potential of unrelated and related cord blood: the first six years of the EUROCORD/NETCORD Bank Germany.

Kögler G, Somville T, Göbel U, Hakenberg P, Knipper A, Fischer J, Adams O, Krempe C, McKenzie C, Rüttgers H, Meier W, Bellmann O, Streng H, Ring A, Rosseck U, Rocha V, Wernet P.

Klin Padiatr. 1999 Jul-Aug;211(4):224-32.

PMID:
10472555
15.

Emerging trends in transplantation of inherited metabolic diseases.

Prasad VK, Kurtzberg J.

Bone Marrow Transplant. 2008 Jan;41(2):99-108. doi: 10.1038/sj.bmt.1705970. Epub 2008 Jan 7. Review.

PMID:
18176609
16.

Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome.

Peters C, Balthazor M, Shapiro EG, King RJ, Kollman C, Hegland JD, Henslee-Downey J, Trigg ME, Cowan MJ, Sanders J, Bunin N, Weinstein H, Lenarsky C, Falk P, Harris R, Bowen T, Williams TE, Grayson GH, Warkentin P, Sender L, Cool VA, Crittenden M, Packman S, Kaplan P, Lockman LA, Anderson J, Krivit W, Dusenbery K, Wagner J.

Blood. 1996 Jun 1;87(11):4894-902.

17.
18.

Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler).

Grigull L, Beilken A, Schrappe M, Das A, Luecke T, Sander A, Stanulla M, Rehe K, Sauer M, Schmid H, Welte K, Lukacs Z, Gal A, Sykora KW.

Bone Marrow Transplant. 2005 Feb;35(3):265-9.

PMID:
15580280
19.

[Transplantation of highly purified CD34+ hematologic peripheral stem cells from haploidentical related donors in the treatment of children with non-malignant diseases].

Starý J, Sedlácek P, Vodvárková S, Poloucková A, Formánková R, Gasová Z, Marinov I.

Cas Lek Cesk. 2002 Mar 29;141(6):176-81. Czech.

PMID:
11977835
20.

The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome.

Krivit W, Lockman LA, Watkins PA, Hirsch J, Shapiro EG.

J Inherit Metab Dis. 1995;18(4):398-412. Review.

PMID:
7494399
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