Transplantation in inborn errors of metabolism: current considerations and future perspectives (Q39151620)

9 September 2017

Transplantation in inborn errors of metabolism: current considerations and future perspectives (English)

1 reference

9 September 2017

1

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31 May 2020

Robert F Wynn

series ordinal

3

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25074667%20AND%20SRC:MED&resulttype=core&format=json

31 May 2020

author name string

Paul J Orchard

series ordinal

2

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31 May 2020

language of work or name

English

0 references

publication date

30 July 2014

1 reference

British Journal of Haematology

9 September 2017

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25074667%20AND%20SRC:MED&resulttype=core&format=json

31 May 2020

volume

167

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31 May 2020

page(s)

293-303

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31 May 2020

issue

3

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The clinical outcome of Hurler syndrome after stem cell transplantation.

31 May 2020

cites work

1 reference

Musculoskeletal manifestations of lysosomal storage disorders.

21 January 2018

1 reference

Body weight-dependent pharmacokinetics of busulfan in paediatric haematopoietic stem cell transplantation patients: towards individualized dosing

21 January 2018

1 reference

Fludarabine and exposure-targeted busulfan compares favorably with busulfan/cyclophosphamide-based regimens in pediatric hematopoietic cell transplantation: maintaining efficacy with less toxicity.

21 January 2018

1 reference

Gene therapy for leukodystrophies

21 January 2018

1 reference

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

21 January 2018

1 reference

Current international perspectives on hematopoietic stem cell transplantation for inherited metabolic disorders.

21 January 2018

1 reference

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning

21 January 2018

1 reference

Regional mapping of the human galactocerebrosidase gene (GALC) to 14q31 by in situ hybridization

21 January 2018

1 reference

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

21 January 2018

1 reference

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

21 January 2018

1 reference

Early infantile Krabbe disease: results of the World-Wide Krabbe Registry.

21 January 2018

1 reference

Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome

21 January 2018

1 reference

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

21 January 2018

1 reference

A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation

21 January 2018

1 reference

Metachromatic leukodystrophy--an update

21 January 2018

1 reference

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

21 January 2018

1 reference

Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a consensus conference proposal for a standardized approach

21 January 2018

1 reference

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

21 January 2018

1 reference

Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature.

21 January 2018

1 reference

Fatty acid abnormality in adrenoleukodystrophy

21 January 2018

1 reference

Enzyme-replacement therapy in mucopolysaccharidosis I.

21 January 2018

1 reference

Weighing the evidence for newborn screening for early-infantile Krabbe disease

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases

21 January 2018

1 reference

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

21 January 2018

1 reference

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

Dangerous and expensive screening and treatment for rare childhood diseases: the case of Krabbe disease

21 January 2018

1 reference

Analysis of MRI patterns aids prediction of progression in X-linked adrenoleukodystrophy

21 January 2018

1 reference

X-linked adrenoleukodystrophy: therapeutic approaches to distinct phenotypes

21 January 2018

1 reference

Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy

21 January 2018

1 reference

Hematopoietic cell therapy for metabolic disease

21 January 2018

1 reference

Pre-transplant risk factors affecting outcome in Hurler syndrome.

21 January 2018

1 reference

Multi-institutional assessments of transplantation for metabolic disorders

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Umbilical cord blood transplantation for juvenile metachromatic leukodystrophy

21 January 2018

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21 January 2018

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Direct demonstration of binding of a lysosomal enzyme, alpha-L-iduronidase, to receptors on cultured fibroblasts

21 January 2018

1 reference

Stem cell transplantation for adult-onset krabbe disease: report of a case

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.

21 January 2018

1 reference

Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB

21 January 2018

1 reference

Busulfan conditioning enhances engraftment of hematopoietic donor-derived cells in the brain compared with irradiation

21 January 2018

1 reference

A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD).

21 January 2018

1 reference

Management of mucopolysaccharidosis type IH (Hurler's syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution's experience

21 January 2018

1 reference

The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I.

21 January 2018

1 reference

21 January 2018

Identifiers

DOI

10.1111/BJH.13059

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25074667%20AND%20SRC:MED&resulttype=core&format=json

31 May 2020

1 reference