The effect of enzyme replacement therapy on clinical outcomes in female patients with Fabry disease – A systematic literature review by a European panel of experts-Reference-Cited by-同舟云学术

The effect of enzyme replacement therapy on clinical outcomes in female patients with Fabry disease – A systematic literature review by a European panel of experts

Published:2019-03 Issue:3 Volume:126 Page:224-235
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Germain Dominique P.,Arad Michael,Burlina Alessandro,Elliott Perry M.,Falissard Bruno,Feldt-Rasmussen Ulla^ORCID,Hilz Max J.,Hughes Derralynn A.,Ortiz Alberto^ORCID,Wanner Christoph,Weidemann Frank,Spada Marco

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference89 articles.

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2. Fabry disease: guidelines for the evaluation and management of multi-organ system involvement;Eng;Genet. Med.,2006

3. Enzyme replacement therapy for Fabry disease: lessons from two alpha-galactosidase A orphan products and one FDA approval;Desnick;Expert. Opin. Biol. Ther.,2004

4. Enzyme replacement therapy for lysosomal diseases: lessons from 20 years of experience and remaining challenges;Desnick;Annu. Rev. Genomics Hum. Genet.,2012

5. Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry;Germain;Genet. Med.,2013

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