Metachromatic leukodystrophy: an overview of current and prospective treatments (Q34870734)

5 August 2017

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title

Metachromatic leukodystrophy: an overview of current and prospective treatments (English)

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metachromatic leukodystrophy

5 August 2017

main subject

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A Biffi

1

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5 August 2017

G Lucchini

series ordinal

2

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5 August 2017

A Rovelli

series ordinal

3

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5 August 2017

M Sessa

series ordinal

4

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5 August 2017

language of work or name

English

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publication date

1 October 2008

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Bone Marrow Transplantation

5 August 2017

published in

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5 August 2017

volume

42 Suppl 2

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5 August 2017

page(s)

S2-6

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The frequency of lysosomal storage diseases in The Netherlands.

5 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

Psychiatric disturbances in metachromatic leukodystrophy. Insights into the neurobiology of psychosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

Lysosomal storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

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%2FBMT.2008.275

7 January 2021

Hematopoietic cell therapy for metabolic disease

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Trends in haematopoietic cell transplantation for inborn errors of metabolism

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Emerging trends in transplantation of inherited metabolic diseases

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

The controversial and changing role of haematopoietic cell transplantation for lysosomal storage disorders: an update

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Enzyme, cell and gene-based therapies for metachromatic leukodystrophy

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Novel candidate disease for gene therapy: metachromatic leukodystrophy

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Molecular basis of different forms of metachromatic leukodystrophy

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Molecular Genetics of Metachromatic Leukodystrophy

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Molecular analysis of the arylsulphatase A gene in late infantile metachromatic leucodystrophy patients and healthy subjects from Italy

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Prevalence of lysosomal storage diseases in Portugal

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

The assay of arylsulphatases A and B in human urine

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Two new polymorphisms in the arylsulfatase A gene and their haplotype associations with normal, metachromatic leukodystrophy and pseudodeficiency alleles

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Practical suggestions in diagnosing metachromatic leukodystrophy in probands and in testing family members

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Identification of nine novel arylsulfatase a (ARSA) gene mutations in patients with metachromatic leukodystrophy (MLD)

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

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%2FBMT.2008.275

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Stabilization of juvenile metachromatic leukodystrophy after bone marrow transplantation: a 13-year follow-up.

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Improved peripheral nerve conduction, EEG and verbal IQ after bone marrow transplantation for adult metachromatic leukodystrophy

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Slow progression of juvenile metachromatic leukodystrophy 6 years after bone marrow transplantation

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Long-term stabilization after bone marrow transplantation in juvenile metachromatic leukodystrophy

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Late juvenile metachromatic leukodystrophy treated with bone marrow transplantation; a 4-year follow-up study

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Early marrow transplantation in a pre-symptomatic neonate with late infantile metachromatic leukodystrophy does not halt disease progression

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Transduction of Human CD34+CD38- Bone Marrow and Cord Blood-Derived SCID-Repopulating Cells with Third-Generation Lentiviral Vectors

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy.

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

1 reference

https://api.crossref.org/works/10.1038%2FBMT.2008.275

7 January 2021

Identifiers

DOI

10.1038/BMT.2008.275

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Dimensions Publication ID

5 August 2017

1029509391

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