(Q35079584)

6 August 2017

1 reference

title

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines (English)

1 reference

6 August 2017

2

C G Steward

1 reference

National Marrow Donor Program

6 August 2017

series ordinal

3

object named as

National Marrow Donor Program

1 reference

6 August 2017

C Peters

1

1 reference

6 August 2017

International Bone Marrow Transplant Registry

series ordinal

4

1 reference

6 August 2017

Working Party on Inborn Errors, European Bone Marrow Transplant Group

series ordinal

5

1 reference

6 August 2017

language of work or name

English

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publication date

1 February 2003

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Bone Marrow Transplantation

6 August 2017

published in

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6 August 2017

volume

31

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6 August 2017

issue

4

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6 August 2017

page(s)

229-239

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The defect in Hurler and Hunter syndromes. II. Deficiency of specific factors involved in mucopolysaccharide degradation

6 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Induced degradation of glycosaminoglycans in Hurler's and Hunter's syndromes by plasma infusion

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Effect of leukocyte transfusion in a child with type II mucopolysaccharidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

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.1703839

Light and electron microscopic features of the liver in mucopolysaccharidosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Resolution of obstructive sleep apnea in Hurler syndrome after bone marrow transplantation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Long-term outcome of Hurler syndrome following bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Surgical treatment of carpal tunnel syndrome and trigger digits in children with mucopolysaccharide storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Outcome of second hematopoietic cell transplantation in Hurler syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

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.1703839

Enzyme-replacement therapy in mucopolysaccharidosis I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Long-term follow-up following bone marrow transplantation for Hunter disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Bone marrow transplantation in Hunter syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Adrenoleukodystrophy: phenotype, genetics, pathogenesis and therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

The importance of testing for adrenoleucodystrophy in males with idiopathic Addison's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Proton MR spectroscopic imaging predicts lesion progression on MRI in X-linked adrenoleukodystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Quantitative proton magnetic resonance spectroscopy of childhood adrenoleukodystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Zellweger syndrome: diagnostic assays, syndrome delineation, and potential therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Peroxisomal and mitochondrial defects in the cerebro-hepato-renal syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Subunits of the translation initiation factor eIF2B are mutant in leukoencephalopathy with vanishing white matter

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

A new leukoencephalopathy with vanishing white matter.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Four year follow-up of a case of fucosidosis treated with unrelated donor bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and S

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Clinical and biochemical outcome of marrow transplantation for Gaucher disease of the Norrbottnian type

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Norrbottnian type of Gaucher disease--clinical, biochemical and molecular biology aspects: successful treatment with bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Allogenic bone marrow transplantation in severe Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Beneficial effect of pre-transplant splenectomy on displacement bone marrow transplantation for Gaucher's syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

A less costly regimen of alglucerase to treat Gaucher's disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Prospective study of neurological responses to treatment with macrophage-targeted glucocerebrosidase in patients with type 3 Gaucher's disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Bone marrow transplantation in late infantile Batten disease and juvenile Batten disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Evidence that lysosomal storage of proteolipids is a cell autonomous process in the motor neuron degeneration (mnd) mouse, a model of neuronal ceroid lipofuscinosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Niemann-Pick disease type C (a cellular cholesterol lipidosis) treated by bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

The heterogeneity of the osteopetroses reflects the diversity of cellular influences during skeletal development

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Recent developments in the understanding of the pathophysiology of osteopetrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Loss of the ClC-7 chloride channel leads to osteopetrosis in mice and man

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Mutations in the a3 subunit of the vacuolar H(+)-ATPase cause infantile malignant osteopetrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Defects in TCIRG1 subunit of the vacuolar proton pump are responsible for a subset of human autosomal recessive osteopetrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Carbonic anhydrase II deficiency in 12 families with the autosomal recessive syndrome of osteopetrosis with renal tubular acidosis and cerebral calcification

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

The association of infantile osteopetrosis and neuronal storage disease in two brothers

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Bone marrow transplantation corrects osteopetrosis in the carbonic anhydrase II deficiency syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Malignant infantile osteopetrosis presenting with neonatal hypocalcaemia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Successful bone-marrow transplantation for infantile malignant osteopetrosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

HLA-haploidentical blood progenitor cell transplantation in osteopetrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Wolman disease successfully treated by bone marrow transplantation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

The p38 mitogen-activated protein kinase pathway plays a critical role in thrombin-induced endothelial chemokine production and leukocyte recruitment

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Allogeneic mesenchymal stem cell infusion for treatment of metachromatic leukodystrophy (MLD) and Hurler syndrome (MPS-IH).

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Bone-Marrow Transplantation in the Maroteaux–Lamy Syndrome (Mucopolysaccharidosis Type VI)

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Ocular changes in the mucopolysaccharidoses after bone marrow transplantation. A preliminary report

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Evaluation of coronary artery disease in the Hurler syndrome by angiography

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Resting Tl-201 scintigraphy in the evaluation of coronary artery disease in children with Hurler syndrome.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Coronary artery patency following long-term successful engraftment 14 years after bone marrow transplantation in the Hurler syndrome

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Follow-up of nine patients with Hurler syndrome after bone marrow transplantation.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Allogeneic bone marrow transplantation for lysosomal storage diseases. The European Group for Bone Marrow Transplantation

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Long-term effects of bone marrow transplantation for inborn errors of metabolism: a study of four patients with lysosomal storage diseases

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Bone marrow transplantation for Sanfilippo disease type B

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

MR spectroscopic imaging of normal-appearing white matter in adrenoleukodystrophy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Treatment of Late Infantile Metachromatic Leukodystrophy by Bone Marrow Transplantation

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Bone marrow-derived mesenchymal stem cells remain host-derived despite successful hematopoietic engraftment after allogeneic transplantation in patients with lysosomal and peroxisomal storage diseases

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Long-term follow-up of the first successful bone marrow transplantation in Gaucher disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Bone-marrow transplantation in severe Gaucher's disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Regression of the radiological changes of Gaucher's disease following bone marrow transplantation

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Bone marrow transplantation for the treatment of alpha-mannosidosis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Bone marrow transplantation for aspartylglucosaminuria: follow-up study of transplanted and non-transplanted patients

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Neuronal ceroid lipofuscinoses in childhood

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Hematopoietic cell transplantation in fetal lambs with ceroid-lipofuscinosis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Batten's disease: failure of allogeneic bone marrow transplantation to arrest disease progression in a canine model.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Biochemical improvement after treatment by bone marrow transplantation in I-cell disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Improvement of tear lysosomal enzyme levels after treatment with bone marrow transplantation in a patient with I-cell disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Bone marrow transplantation in canine GM1 gangliosidosis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Cells that resorb bone

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Bone marrow transplantation for autosomal recessive osteopetrosis. A report from the Working Party on Inborn Errors of the European Bone Marrow Transplantation Group

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Hematopoietic stem cell transplantation for infantile osteopetrosis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Allogeneic bone-marrow transplantation in infantile malignant osteopetrosis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Bone-marrow transplantation for immunodeficiencies and osteopetrosis: European survey, 1968-1985

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Imaging of malignant infantile osteopetrosis before and after bone marrow transplantation

1 reference

https://api.crossref.org/works/10.1038%2FSJ.BMT.1703839

7 January 2021

Identifiers

DOI

10.1038/SJ.BMT.1703839

1 reference

Dimensions Publication ID

6 August 2017

1028761877

0 references

1 reference