(Q34389992)

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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

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Europe PubMed Central

PubMed publication ID

1 August 2017

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 (English)

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

0 references

enzyme replacement therapy

0 references

Vassili Valayannopoulos

10

object named as

Vassili Valayannopoulos

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

8

object named as

Eugen Mengel

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Roberto Giugliani

11

object named as

Roberto Giugliani

1 reference

Europe PubMed Central

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1 August 2017

author name string

Christian J Hendriksz

1

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Europe PubMed Central

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1 August 2017

Barbara Burton

2

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Europe PubMed Central

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1 August 2017

Thomas R Fleming

3

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Paul Harmatz

4

1 reference

Europe PubMed Central

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1 August 2017

Derralynn Hughes

5

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Europe PubMed Central

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1 August 2017

Simon A Jones

6

1 reference

Europe PubMed Central

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1 August 2017

Shuan-Pei Lin

7

1 reference

Europe PubMed Central

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1 August 2017

Maurizio Scarpa

9

1 reference

Europe PubMed Central

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1 August 2017

STRIVE Investigators

12

1 reference

Europe PubMed Central

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1 August 2017

Peter Slasor

13

1 reference

Europe PubMed Central

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1 August 2017

Debra Lounsbury

14

1 reference

Europe PubMed Central

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1 August 2017

Wolfgang Dummer

15

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Europe PubMed Central

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1 August 2017

publication date

9 May 2014

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Europe PubMed Central

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1 August 2017

Journal of Inherited Metabolic Disease

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Europe PubMed Central

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1 August 2017

37

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Europe PubMed Central

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1 August 2017

6

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Europe PubMed Central

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1 August 2017

979-990

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

The 6-Minute Walk Test and Person-Reported Outcomes in Boys with Duchenne Muscular Dystrophy and Typically Developing Controls: Longitudinal Comparisons and Clinically-Meaningful Changes Over One Year

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

Subjective and Objective Assessment of Hand Function in Mucopolysaccharidosis IVa Patients

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

Current and emerging management options for patients with Morquio A syndrome

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

Biomarkers and surrogate endpoints in clinical trials

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

Clinical utility of endurance measures for evaluation of treatment in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

Enzyme Replacement in a Human Model of Mucopolysaccharidosis IVA In Vitro and Its Biodistribution in the Cartilage of Wild Type Mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

Incidence of the mucopolysaccharidoses in Northern Ireland

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

A comparison of continuous intravenous epoprostenol (prostacyclin) with conventional therapy for primary pulmonary hypertension

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

Standardization of Spirometry, 1994 Update. American Thoracic Society

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

Carbimazole and exercise tolerance in chronic airflow obstruction

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4206772

6 July 2018

Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9715-6

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9715-6

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9715-6

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9715-6

21 January 2018

A sharper Bonferroni procedure for multiple tests of significance

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9715-6

21 January 2018

Standard reference for the six-minute-walk test in healthy children aged 7 to 16 years.

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9715-6

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9715-6

21 January 2018

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9715-6

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1007%2FS10545-014-9715-6

21 January 2018

Incidence of the mucopolysaccharidoses in Western Australia

1 reference

https://pubmed.ncbi.nlm.nih.gov/24810369

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/24810369

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Interpreting small differences in functional status: the Six Minute Walk test in chronic lung disease patients

1 reference

https://pubmed.ncbi.nlm.nih.gov/24810369

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

1 reference

https://pubmed.ncbi.nlm.nih.gov/24810369

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

https://pubmed.ncbi.nlm.nih.gov/24810369

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

The 6-minute walk test: normal values for children of 4-11 years of age

1 reference

https://pubmed.ncbi.nlm.nih.gov/24810369

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1007/S10545-014-9715-6

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

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0 references

PMC publication ID

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

ResearchGate publication ID

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