(Q45118891)

9 December 2017

Characterization of Fabry disease in 352 pediatric patients in the Fabry Registry (English)

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9 December 2017

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4

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9 December 2017

8

Anna Tylki-Szymanska

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9 December 2017

Dominique P Germain

6

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John J Bissler

object named as

John Bissler

2

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Robert J. Hopkin

object named as

Robert J Hopkin

1

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author name string

Maryam Banikazemi

3

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9 December 2017

Christine M Eng

5

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9 December 2017

Roberta Lemay

7

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9 December 2017

William R Wilcox

9

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9 December 2017

language of work or name

English

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publication date

1 November 2008

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9 December 2017

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9 December 2017

64

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9 December 2017

5

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9 December 2017

550-555

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https://scigraph.springernature.com/pub.10.1203/pdr.0b013e318183f132

9 December 2017

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cites work

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

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

A phase 1/2 clinical trial of enzyme replacement in fabry disease: pharmacokinetic, substrate clearance, and safety studies

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

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/10.1203%2FPDR.0B013E318183F132

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

Safety and efficacy of enzyme replacement therapy with agalsidase beta: an international, open-label study in pediatric patients with Fabry disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

21 January 2018

Pediatric Fabry disease

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

Methods for validating and norming translations of health status questionnaires: the IQOLA Project approach. International Quality of Life Assessment.

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

Gastrointestinal symptoms in 342 patients with Fabry disease: prevalence and response to enzyme replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

National Kidney Foundation's Kidney Disease Outcomes Quality Initiative clinical practice guidelines for chronic kidney disease in children and adolescents: evaluation, classification, and stratification

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

Using standardized serum creatinine values in the modification of diet in renal disease study equation for estimating glomerular filtration rate

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2FPDR.0B013E318183F132

10.1203/PDR.0B013E318183F132

21 January 2018

Identifiers

DOI

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Dimensions Publication ID

9 December 2017

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1 reference