Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome. (Q35319254)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25897204%20AND%20SRC:MED&resulttype=core&format=json

6 March 2020

review article

2 references

3 July 2021

Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25897204%20AND%20SRC:MED&resulttype=core&format=json

enzyme replacement therapy

6 March 2020

main subject

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hematopoietic stem cell transplantation

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3 July 2021

Carlos J Alméciga-Díaz

3

1 reference

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Michael B Bober

5

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Alexander Rodríguez-López

6 March 2020

12

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6 March 2020

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Roberto Giugliani

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6 March 2020

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Shunji Tomatsu

1

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6 March 2020

Kazuki Sawamoto

2

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6 March 2020

Tsutomu Shimada

4

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6 March 2020

Yasutsugu Chinen

6

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Hiromasa Yabe

7

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Adriana M Montaño

8

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Francyne Kubaski

10

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Eriko Yasuda

11

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Angela J Espejo-Mojica

13

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Oscar F Sánchez

14

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Robert W Mason

15

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Luis A Barrera

16

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William G Mackenzie

17

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Tadao Orii

18

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0 references

publication date

1 April 2015

10 references

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6 March 2020

3 July 2021

Drug Design, Development and Therapy

10 references

12 March 2019

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17 June 2019

25 June 2019

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1937-1953

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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12 July 2018

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Outcomes of haematopoietic stem cell transplantation for inherited metabolic disorders: a report from the Australian and New Zealand Children's Haematology Oncology Group and the Australasian Bone Marrow Transplant Recipient Registry

12 July 2018

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

26 September 2018

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26 September 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

The Morquio A Clinical Assessment Program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Anesthetic care and perioperative complications of children with Morquio syndrome.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Enzyme-replacement therapy in life-threatening hypophosphatasia.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Enzyme replacement therapy for Morquio A: an active recombinant N-acetylgalactosamine-6-sulfate sulfatase produced in Escherichia coli BL21.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Localized donor cells in brain of a Hunter disease patient after cord blood stem cell transplantation.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Characterization and pharmacokinetic study of recombinant human N-acetylgalactosamine-6-sulfate sulfatase.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Development and testing of new screening method for keratan sulfate in mucopolysaccharidosis IVA.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Musculoskeletal manifestations of Hurler syndrome: long-term follow-up after bone marrow transplantation

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Mouse model of N-acetylgalactosamine-6-sulfate sulfatase deficiency (Galns-/-) produced by targeted disruption of the gene defective in Morquio A disease

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Prognostic usefulness of the six-minute walk in patients with advanced congestive heart failure secondary to ischemic or nonischemic cardiomyopathy.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Novel drug delivery system to bone using acidic oligopeptide: pharmacokinetic characteristics and pharmacological potential

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Enzyme therapy for lysosomal acid lipase deficiency in the mouse

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Nonablative neonatal marrow transplantation attenuates functional and physical defects of beta-glucuronidase deficiency.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Enzyme-replacement therapy in mucopolysaccharidosis I.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Osteotropic drug delivery system (ODDS) based on bisphosphonic prodrug. I.v. effects of osteotropic estradiol on bone mineral density and uterine weight in ovariectomized rats

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4389814

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

24 October 2018

1 reference

12 December 2020

Validation of keratan sulfate level in mucopolysaccharidosis type IVA by liquid chromatography-tandem mass spectrometry

1 reference

12 December 2020

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

1 reference

12 December 2020

The presenting features of mucopolysaccharidosis type IH (Hurler syndrome)

1 reference

12 December 2020

Validation of an LC-MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome

1 reference

12 December 2020

Long-term follow-up of a girl with Maroteaux-Lamy syndrome after bone marrow transplantation

1 reference

12 December 2020

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

1 reference

12 December 2020

Bone-marrow transplantation in Hurler's syndrome. Effect on skeletal development

1 reference

12 December 2020

First pilot newborn screening for four lysosomal storage diseases in an Italian region: identification and analysis of a putative causative mutation in the GBA gene

1 reference

12 December 2020

Hip dysplasia in Hurler's syndrome: orthopaedic management after bone marrow transplantation

1 reference

12 December 2020

Analysis of factors affecting development of carpal tunnel syndrome in patients with Hurler syndrome after hematopoietic cell transplantation

1 reference

12 December 2020

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

1 reference

12 December 2020

Fetal presentation of Morquio disease type A

1 reference

12 December 2020

Identifiers

DOI

10.2147/DDDT.S68562

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25897204%20AND%20SRC:MED&resulttype=core&format=json

6 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25897204%20AND%20SRC:MED&resulttype=core&format=json

6 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25897204%20AND%20SRC:MED&resulttype=core&format=json