(Q50856686)

24 March 2018

title

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

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24 March 2018

author

Jakub Tolar

1

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24 March 2018

author name string

Grewal SS

2

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24 March 2018

Bjoraker KJ

3

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24 March 2018

Whitley CB

4

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24 March 2018

Shapiro EG

5

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24 March 2018

Charnas L

6

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24 March 2018

Orchard PJ

7

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24 March 2018

language of work or name

English

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publication date

26 November 2007

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24 March 2018

Bone Marrow Transplantation

published in

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24 March 2018

volume

41

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24 March 2018

issue

6

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24 March 2018

page(s)

531-535

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24 March 2018

https://scigraph.springernature.com/pub.10.1038/sj.bmt.1705934

exact match

0 references

cites work

The defect in the Hurler and Scheie syndromes: deficiency of -L-iduronidase

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

Trends in haematopoietic cell transplantation for inborn errors of metabolism

21 January 2018

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The defect in Hurler's and Hunter's syndromes: faulty degradation of mucopolysaccharide

21 January 2018

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Enzyme-replacement therapy in mucopolysaccharidosis I.

21 January 2018

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Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

Long-term outcome of Hurler syndrome following bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

Inflammatory cytokines and the development of pulmonary complications after allogeneic hematopoietic cell transplantation in patients with inherited metabolic storage disorders.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

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

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

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

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

Oral busulfan pharmacokinetics and engraftment in children with Hurler syndrome and other inherited metabolic storage diseases undergoing hematopoietic cell transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

Pre-stem cell transplantation enzyme replacement therapy in Hurler syndrome does not lead to significant antibody formation or delayed recovery of the endogenous enzyme post-transplant: a case report.

21 January 2018

1 reference

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A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years.

21 January 2018

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Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources

21 January 2018

1 reference

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Outcome of second hematopoietic cell transplantation in Hurler syndrome.

21 January 2018

1 reference

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Preformed antibody, not primed T cells, is the initial and major barrier to bone marrow engraftment in allosensitized recipients

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

Marrow stromal cells from patients affected by MPS I differentially support haematopoietic progenitor cell development

21 January 2018

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Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts

21 January 2018

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7 January 2021

Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

7 January 2021

Measurements from normal umbilical cord blood of four lysosomal enzymatic activities: alpha-L-iduronidase (Hurler), galactocerebrosidase (globoid cell leukodystrophy), arylsulfatase A (metachromatic leukodystrophy), arylsulfatase B (Maroteaux-Lamy)

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https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

7 January 2021

Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors

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https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

7 January 2021

IBMTR Severity Index for grading acute graft-versus-host disease: retrospective comparison with Glucksberg grade

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

7 January 2021

Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1705934

7 January 2021

Identifiers

DOI

10.1038/SJ.BMT.1705934

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24 March 2018

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24 March 2018