Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA. (Q34938821)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25593792%20AND%20SRC:MED&resulttype=core&format=json

28 February 2020

title

Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA (English)

1 reference

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28 February 2020

1 reference

1 reference

3

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28 February 2020

author name string

Yasutsugu Chinen

1

1 reference

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28 February 2020

Takeshi Higa

2

1 reference

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28 February 2020

Yasuyuki Suzuki

4

1 reference

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28 February 2020

Tadao Orii

5

1 reference

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28 February 2020

Nobuyuki Hyakuna

6

1 reference

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28 February 2020

publication date

1 January 2014

1 reference

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Molecular Genetics and Metabolism Reports

28 February 2020

published in

1 reference

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28 February 2020

volume

1

1 reference

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28 February 2020

page(s)

31-41

1 reference

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Current and emerging treatments and surgical interventions for Morquio A syndrome: a review

28 February 2020

cites work

1 reference

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Review of clinical presentation and diagnosis of mucopolysaccharidosis IVA

8 July 2018

1 reference

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Mucopolysaccharidosis type IVA (Morquio A disease): clinical review and current treatment

8 July 2018

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Adeno-associated virus gene transfer in Morquio A disease - effect of promoters and sulfatase-modifying factor 1.

8 July 2018

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Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.

8 July 2018

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Treatment of lysosomal storage disorders : progress with enzyme replacement therapy

8 July 2018

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Hematopoietic cell therapy for metabolic disease

8 July 2018

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Nanomedicine: clinical applications of polyethylene glycol conjugated proteins and drugs

8 July 2018

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A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

8 July 2018

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A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1.

8 July 2018

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Cell and gene-based therapies for the lysosomal storage diseases

8 July 2018

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Characterizing biological products and assessing comparability following manufacturing changes.

8 July 2018

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

8 July 2018

1 reference

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Gene therapy for lysosomal storage diseases: the lessons and promise of animal models

8 July 2018

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

8 July 2018

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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

8 July 2018

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Pathogenesis of Morquio A syndrome: an autopsied case reveals systemic storage disorder.

8 July 2018

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Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: a nationwide survey in Japan

26 October 2018

1 reference

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Anesthetic care and perioperative complications of children with Morquio syndrome

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4292891

Idursulfase treatment of Hunter syndrome in children younger than 6 years: results from the Hunter Outcome Survey

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4292891

Growth pattern and growth prediction of body height in children with mucopolysaccharidosis type II.

26 October 2018

1 reference

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Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 years

26 October 2018

1 reference

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Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4292891

Growth charts for patients affected with Morquio A disease.

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4292891

Enzyme replacement therapy in a murine model of Morquio A syndrome.

26 October 2018

1 reference

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International Morquio A Registry: clinical manifestation and natural course of Morquio A disease.

26 October 2018

1 reference

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Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-ac

26 October 2018

1 reference

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Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).

26 October 2018

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Enzyme-replacement therapy in mucopolysaccharidosis I.

26 October 2018

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

26 October 2018

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Sacral dimple: incidental findings from newborn evaluation. Mucopolysaccharidosis IVA disease

26 October 2018

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The enzymic defects in Morquio and Maroteaux-Lamy syndrome

26 October 2018

1 reference

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Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, o

7 January 2021

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

7 January 2021

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Morquio disease: The role of cartilage canals in the pathogenesis of chondrogenic dwarfism

7 January 2021

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGMR.2013.11.002

Prospective study of 11 Brazilian patients with mucopolysaccharidosis II

7 January 2021

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25593792

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Occipito-atlanto-axial fusion in Morquio-Brailsford syndrome. A ten-year experience

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25593792

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1016/J.YMGMR.2013.11.002

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25593792%20AND%20SRC:MED&resulttype=core&format=json

28 February 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25593792%20AND%20SRC:MED&resulttype=core&format=json

28 February 2020

PubMed publication ID

25593792

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25593792%20AND%20SRC:MED&resulttype=core&format=json