Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease (Q35550696)

8 August 2017

title

Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease (English)

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enzyme replacement therapy

8 August 2017

main subject

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Dominique P Germain

1

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Robin Lachmann

object named as

Robin H Lachmann

6

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David G Warnock

12

object named as

David G Warnock

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8 August 2017

3

Robert J Desnick

1 reference

8 August 2017

13

Neal J Weinreb

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8 August 2017

8

Gabor E Linthorst

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8 August 2017

2

Joel Charrow

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8 August 2017

Nathalie Guffon

4

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Judy Kempf

5

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8 August 2017

Roberta Lemay

7

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8 August 2017

Seymour Packman

9

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8 August 2017

C Ronald Scott

10

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Stephen Waldek

11

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William R Wilcox

14

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language of work or name

English

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publication date

20 March 2015

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Journal of Medical Genetics

8 August 2017

published in

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8 August 2017

volume

52

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8 August 2017

issue

5

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8 August 2017

page(s)

353-358

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Natural course of Fabry disease and the effectiveness of enzyme replacement therapy: a systematic review and meta-analysis: effectiveness of ERT in different disease stages

8 August 2017

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Long-term outcome of enzyme-replacement therapy in advanced Fabry disease: evidence for disease progression towards serious complications

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Agalsidase benefits renal histology in young patients with Fabry disease

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

End-stage renal disease in patients with Fabry disease: natural history data from the Fabry Registry

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Fabry disease: progression of nephropathy, and prevalence of cardiac and cerebrovascular events before enzyme replacement therapy

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Stroke in Fabry disease frequently occurs before diagnosis and in the absence of other clinical events: natural history data from the Fabry Registry

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Recommendations and guidelines for the diagnosis and treatment of Fabry nephropathy in adults

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Fabry disease: guidelines for the evaluation and management of multi-organ system involvement

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Long-term safety and efficacy of enzyme replacement therapy for Fabry disease

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Angiokeratoma corporis diffusum--Fabry's disease

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Longitudinal studies on the rate of decline in renal function with age.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Females with Fabry disease frequently have major organ involvement: lessons from the Fabry Registry.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Progression risk, urinary protein excretion, and treatment effects of angiotensin-converting enzyme inhibitors in nondiabetic kidney 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=4413801

The Dutch Fabry cohort: diversity of clinical manifestations and Gb3 levels.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4413801

26 September 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102797

Agalsidase-beta therapy for advanced Fabry disease: a randomized trial

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25795794

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Renal biopsy findings in children and adolescents with Fabry disease and minimal albuminuria

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25795794

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1136/JMEDGENET-2014-102797

1 reference

8 August 2017

1 reference

8 August 2017

1 reference