(Q51646061)

8 April 2018

title

A 4-year study of the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa in 36 women with Fabry disease. (English)

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8 April 2018

enzyme replacement therapy

main subject

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3

Eugen Mengel

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8 April 2018

Catharina Whybra

1

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8 April 2018

Elke Miebach

2

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8 April 2018

Andreas Gal

4

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8 April 2018

Karin Baron

5

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8 April 2018

Michael Beck

6

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8 April 2018

Christoph Kampmann

7

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8 April 2018

language of work or name

English

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publication date

1 June 2009

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8 April 2018

published in

Genetics in Medicine

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8 April 2018

volume

11

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8 April 2018

issue

6

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8 April 2018

page(s)

441-449

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8 April 2018

https://scigraph.springernature.com/pub.10.1097/gim.0b013e3181a23bec

exact match

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cites work

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1 reference

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21 January 2018

Pediatric Fabry disease

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The early clinical phenotype of Fabry disease: a study on 35 European children and adolescents.

21 January 2018

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The heart in Anderson Fabry disease

21 January 2018

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Prevalence of Fabry disease in patients with cryptogenic stroke: a prospective study.

21 January 2018

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Fabry disease: impaired autonomic function.

21 January 2018

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Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease.

21 January 2018

1 reference

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Cardiac manifestations of Anderson-Fabry disease in children and adolescents

21 January 2018

1 reference

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Natural history of Fabry renal disease: influence of alpha-galactosidase A activity and genetic mutations on clinical course

21 January 2018

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Prevalence of lysosomal storage disorders

21 January 2018

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High incidence of later-onset fabry disease revealed by newborn screening

21 January 2018

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The pattern of inheritance of X-linked traits is not dominant or recessive, just X-linked

21 January 2018

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

21 January 2018

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Heterozygous Fabry women are not just carriers, but have a significant burden of disease and impaired quality of life.

21 January 2018

1 reference

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Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 60 obligate carrier females

21 January 2018

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Natural history of Fabry disease in females in the Fabry Outcome Survey.

21 January 2018

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Clinical presentation in female patients with Fabry disease

21 January 2018

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Clinical manifestations of Fabry disease in children: data from the Fabry Outcome Survey.

21 January 2018

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Characterization of Fabry disease in 352 pediatric patients in the Fabry Registry

21 January 2018

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

21 January 2018

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21 January 2018

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Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey

21 January 2018

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

21 January 2018

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Fabry disease: baseline medical characteristics of a cohort of 1765 males and females in the Fabry Registry.

21 January 2018

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The Mainz Severity Score Index: a new instrument for quantifying the Anderson-Fabry disease phenotype, and the response of patients to enzyme replacement therapy.

21 January 2018

1 reference

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Comparison of health-related quality of life between heterozygous women with Fabry disease, a healthy control population, and patients with other chronic disease.

21 January 2018

1 reference

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Quality of life of patients with Fabry disease.

21 January 2018

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Assessment of health-related quality-of-life in males with Anderson Fabry Disease before therapeutic intervention

21 January 2018

1 reference

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Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease

21 January 2018

1 reference

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Enzyme replacement therapy in Fabry disease: a randomized controlled trial

21 January 2018

1 reference

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Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting

21 January 2018

1 reference

https://api.crossref.org/works/10.1097%2FGIM.0B013E3181A23BEC

Enzyme replacement therapy in heterozygous females with Fabry disease: results of a phase IIIB study

21 January 2018

1 reference

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Clinical practice. Nondiabetic kidney disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1097%2FGIM.0B013E3181A23BEC

21 January 2018

1 reference

https://api.crossref.org/works/10.1097%2FGIM.0B013E3181A23BEC

Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing.

21 January 2018

1 reference

https://api.crossref.org/works/10.1097%2FGIM.0B013E3181A23BEC

Functional class in patients with heart failure is associated with the development of diabetes

21 January 2018

1 reference

https://api.crossref.org/works/10.1097%2FGIM.0B013E3181A23BEC

Nephropathy in males and females with Fabry disease: cross-sectional description of patients before treatment with enzyme replacement therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1097%2FGIM.0B013E3181A23BEC

Early renal changes in hemizygous and heterozygous patients with Fabry's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1097%2FGIM.0B013E3181A23BEC

Fabry disease: overall effects of agalsidase alfa treatment

21 January 2018

1 reference

https://api.crossref.org/works/10.1097%2FGIM.0B013E3181A23BEC

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1097%2FGIM.0B013E3181A23BEC

Cardiac manifestations of Anderson-Fabry disease in heterozygous females

21 January 2018

1 reference

https://api.crossref.org/works/10.1097%2FGIM.0B013E3181A23BEC

10.1097/GIM.0B013E3181A23BEC

21 January 2018

Identifiers

DOI

1 reference

8 April 2018

Dimensions Publication ID

0 references

1 reference

8 April 2018