(Q36677856)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

title

Enzyme replacement therapy for treating mucopolysaccharidosis type IVA (Morquio A syndrome): effect and limitations (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

enzyme replacement therapy

10 March 2020

main subject

0 references

mucopolysaccharidosis type IVA

1 reference

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

Carlos J Alméciga-Díaz

9

1 reference

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10 March 2020

author name string

Shunji Tomatsu

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

Kazuki Sawamoto

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

Tsutomu Shimada

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

Francyne Kubaski

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

Eriko Yasuda

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

Robert W Mason

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

Shaukat Khan

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

Luis A Barrera

10

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

William G Mackenzie

11

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

Tadao Orii

12

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

publication date

29 October 2015

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

Expert opinion on orphan drugs

10 March 2020

published in

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

volume

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

issue

11

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

page(s)

1279-1290

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

Human recombinant lysosomal enzymes produced in microorganisms.

10 March 2020

cites work

1 reference

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

30 September 2017

1 reference

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

30 September 2017

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Therapies for the bone in mucopolysaccharidoses

30 September 2017

1 reference

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Activities of daily living in patients with Hunter syndrome: impact of enzyme replacement therapy and hematopoietic stem cell transplantation

30 September 2017

1 reference

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Therapies of mucopolysaccharidosis IVA (Morquio A syndrome).

30 September 2017

1 reference

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Morquio A syndrome: diagnosis and current and future therapies

30 September 2017

1 reference

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Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial

30 September 2017

1 reference

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Pharmacokinetic and pharmacodynamic evaluation of elosulfase alfa, an enzyme replacement therapy in patients with Morquio A syndrome

30 September 2017

1 reference

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Impact of Enzyme Replacement Therapy and Hematopoietic Stem Cell Therapy on Growth in Patients with Hunter Syndrome.

30 September 2017

1 reference

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Growth charts for patients with Hunter syndrome.

30 September 2017

1 reference

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Enzyme replacement therapy in newborn mucopolysaccharidosis IVA mice: early treatment rescues bone lesions?

30 September 2017

1 reference

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Chondroitin 6-Sulfate as a Novel Biomarker for Mucopolysaccharidosis IVA and VII.

30 September 2017

1 reference

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

30 September 2017

1 reference

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Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

30 September 2017

1 reference

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Enzyme replacement therapy on hypophosphatasia mouse model

30 September 2017

1 reference

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Mucopolysaccharidosis IVA: correlation between genotype, phenotype and keratan sulfate levels

30 September 2017

1 reference

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Newborn screening and diagnosis of mucopolysaccharidoses

30 September 2017

1 reference

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Neonatal Bone Marrow Transplantation in MPS IIIA Mice

30 September 2017

1 reference

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Long circulating enzyme replacement therapy rescues bone pathology in mucopolysaccharidosis VII murine model

30 September 2017

1 reference

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Research priorities in biomarkers and surrogate end-points

30 September 2017

1 reference

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Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA

30 September 2017

1 reference

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

30 September 2017

1 reference

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Enzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type mice

30 September 2017

1 reference

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Skeletal dysplasias: evaluation with impulse oscillometry and thoracoabdominal motion analysis

30 September 2017

1 reference

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Enhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome.

30 September 2017

1 reference

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

30 September 2017

1 reference

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Acidic amino acid tag enhances response to enzyme replacement in mucopolysaccharidosis type VII mice.

30 September 2017

1 reference

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Enzyme replacement therapy for murine hypophosphatasia

30 September 2017

1 reference

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Treatment of lysosomal storage disorders : progress with enzyme replacement therapy

30 September 2017

1 reference

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Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy

30 September 2017

1 reference

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Analytical method for determination of disaccharides derived from keratan sulfates in human serum and plasma by high-performance liquid chromatography/turbo-ionspray ionization tandem mass spectrometry

30 September 2017

1 reference

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Nanomedicine: clinical applications of polyethylene glycol conjugated proteins and drugs

30 September 2017

1 reference

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

30 September 2017

1 reference

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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.

30 September 2017

1 reference

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Enhancement of drug delivery to bone: characterization of human tissue-nonspecific alkaline phosphatase tagged with an acidic oligopeptide

30 September 2017

1 reference

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Biomarkers and surrogate endpoints

30 September 2017

1 reference

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Characterizing biological products and assessing comparability following manufacturing changes.

30 September 2017

1 reference

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Enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome): a preliminary report

30 September 2017

1 reference

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Selective delivery of estradiol to bone by aspartic acid oligopeptide and its effects on ovariectomized mice

30 September 2017

1 reference

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Selective drug delivery system to bone: small peptide (Asp)6 conjugation

30 September 2017

1 reference

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Biochemical defect of non-keratan-sulfate-excreting Morquio syndrome

30 September 2017

1 reference

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The primary structure of a cell-binding bone sialoprotein

30 September 2017

1 reference

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Heterogeneity of Morquio disease.

30 September 2017

1 reference

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The nature and significance of osteopontin

30 September 2017

1 reference

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A sensitive procedure for the diagnosis of N-acetyl-galactosamine-6-sulfate sulfatase deficiency in classical Morquio's disease

30 September 2017

1 reference

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Oxidative stress and inflammation in mucopolysaccharidosis type IVA patients treated with enzyme replacement therapy.

30 September 2017

1 reference

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Comparison of liquid chromatography-tandem mass spectrometry and sandwich ELISA for determination of keratan sulfate in plasma and urine

30 September 2017

1 reference

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Biosynthesis of bone proteins [SPP-1 (secreted phosphoprotein-1, osteopontin), BSP (bone sialoprotein) and SPARC (osteonectin)] in association with mineralized-tissue formation by fetal-rat calvarial cells in culture

28 June 2018

1 reference

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

28 June 2018

1 reference

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

5 September 2018

1 reference

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Anesthetic care and perioperative complications of children with Morquio syndrome.

5 September 2018

1 reference

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Enzyme-replacement therapy in life-threatening hypophosphatasia.

5 September 2018

1 reference

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Six-minute walking test and the assessment of cardiorespiratory responses during weight-loss programmes in obese children

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4788508

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

5 September 2018

1 reference

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The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy.

5 September 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

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4788508

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

5 September 2018

1 reference

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

5 September 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

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4788508

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

5 September 2018

1 reference

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

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4788508

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

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4788508

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

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4788508

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

5 September 2018

1 reference

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

5 September 2018

1 reference

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Enzyme-replacement therapy in mucopolysaccharidosis I.

5 September 2018

1 reference

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Clinical correlates and prognostic significance of six-minute walk test in patients with primary pulmonary hypertension. Comparison with cardiopulmonary exercise testing.

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4788508

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

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4788508

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

5 September 2018

1 reference

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

5 September 2018

1 reference

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

20 October 2018

1 reference

12 December 2020

Fetal presentation of Morquio disease type A

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

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

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

The enzymic defects in Morquio and Maroteaux-Lamy syndrome

1 reference

12 December 2020

The six-minute walking test in children with cystic fibrosis: reliability and validity

1 reference

12 December 2020

10.1517/21678707.2015.1086640

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26973801%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

1 reference