(Q35903551)

11 August 2017

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title

Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases (English)

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11 August 2017

William Krivit

1

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11 August 2017

25 September 2004

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Seminars in Immunopathology

11 August 2017

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11 August 2017

26

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11 August 2017

1-2

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11 August 2017

119-132

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https://scigraph.springernature.com/pub.10.1007/s00281-004-0166-2

11 August 2017

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cites work

A genetic model of substrate deprivation therapy for a glycosphingolipid storage disorder

1 reference

12 December 2020

Tandem mass spectrometry for the direct assay of enzymes in dried blood spots: application to newborn screening for Krabbe disease

1 reference

12 December 2020

Bone marrow transplantation for infantile ceramidase deficiency (Farber disease).

1 reference

12 December 2020

Substrate reduction therapy for glycosphingolipid storage disorders

1 reference

12 December 2020

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

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12 December 2020

Hypertrophic cardiomyopathy in mucopolysaccharidoses: regression after bone marrow transplantation

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12 December 2020

Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis.

1 reference

12 December 2020

Bone marrow transplantation in mucopolysaccharidosis type IIIA: a comparison of an early treated patient with his untreated sibling

1 reference

12 December 2020

Diagnosis of lysosomal storage disorders: evaluation of lysosome-associated membrane protein LAMP-1 as a diagnostic marker

1 reference

12 December 2020

White matter dysfunction and its neuropsychological correlates: a longitudinal study of a case of metachromatic leukodystrophy treated with bone marrow transplant.

1 reference

12 December 2020

Substrate-reduction therapy enhances the benefits of bone marrow transplantation in young mice with globoid cell leukodystrophy

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12 December 2020

Tauroursodeoxycholic acid, a bile acid, is neuroprotective in a transgenic animal model of Huntington's disease.

1 reference

12 December 2020

Stem cell bone marrow transplantation in patients with metabolic storage diseases.

1 reference

12 December 2020

Amelioration of clinical disease following bone marrow transplantation in fucosidase-deficient dogs.

1 reference

12 December 2020

Globoid cell leukodystrophy (Krabbe disease): normal umbilical cord blood galactocerebrosidase activity and polymorphic mutations

1 reference

12 December 2020

Bone marrow transplantation for the treatment of alpha-mannosidosis

1 reference

12 December 2020

Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999.

1 reference

12 December 2020

Twitcher mice with only a single active galactosylceramide synthase gene exhibit clearly detectable but therapeutically minor phenotypic improvements

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12 December 2020

Correction of odontoid dysplasia following bone-marrow transplantation and engraftment (in Hurler syndrome MPS 1H).

1 reference

12 December 2020

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

1 reference

12 December 2020

Correction of feline arylsulphatase B deficiency (mucopolysaccharidosis VI) by bone marrow transplantation

1 reference

12 December 2020

X-Linked adrenoleukodystrophy: overview and prognosis as a function of age and brain magnetic resonance imaging abnormality. A study involving 372 patients

1 reference

12 December 2020

Prevalence of lysosomal storage disorders

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12 December 2020

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

1 reference

12 December 2020

Evaluation of white matter anisotropy in Krabbe disease with diffusion tensor MR imaging: initial experience

1 reference

12 December 2020

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

1 reference

12 December 2020

Leukodystrophies: pathogenesis, diagnosis, strategies, therapies, and future research directions

1 reference

12 December 2020

Usefulness of bone marrow transplantation in the Hurler syndrome

1 reference

12 December 2020

Enzyme replacement therapy in mucopolysaccharidosis I: altered distribution and targeting of alpha-L-iduronidase in immunized rats

1 reference

12 December 2020

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

1 reference

12 December 2020

Evaluation of the preventive effect of glyceryl trioleate-trierucate ("Lorenzo's oil") therapy in X-linked adrenoleukodystrophy: results of two concurrent trials

1 reference

12 December 2020

Evolution of phenotypes in adult male patients with X-linked adrenoleukodystrophy

1 reference

12 December 2020

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)

1 reference

12 December 2020

Molecular heterogeneity of late-onset forms of globoid-cell leukodystrophy

1 reference

12 December 2020

Adrenoleukodystrophy: incidence, new mutation rate, and results of extended family screening

1 reference

12 December 2020

Gentamicin-mediated suppression of Hurler syndrome stop mutations restores a low level of alpha-L-iduronidase activity and reduces lysosomal glycosaminoglycan accumulation

1 reference

12 December 2020

Hematopoietic cell transplantation for mucopolysaccharidosis IIB (Hunter syndrome).

1 reference

12 December 2020

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

1 reference

12 December 2020

Wolman's disease: a review of treatment with bone marrow transplantation and considerations for the future

1 reference

12 December 2020

Wolman disease successfully treated by bone marrow transplantation.

1 reference

12 December 2020

Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group

1 reference

12 December 2020

Late juvenile-onset Krabbe's disease

1 reference

12 December 2020

Effective treatment of alpha-mannosidosis by allogeneic hematopoietic stem cell transplantation.

1 reference

12 December 2020

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

1 reference

12 December 2020

Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy

1 reference

12 December 2020

Allogeneic bone marrow transplantation for Niemann-Pick disease (type IA)

1 reference

12 December 2020

Stemming the tide: glycosphingolipid synthesis inhibitors as therapy for storage diseases

1 reference

12 December 2020

10.1007/S00281-004-0166-2

Identifiers

DOI

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11 August 2017

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PubMed ID

15452666