Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease
Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease
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Publisher
England: BMJ Publishing Group LTD
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Language
English
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Publisher
England: BMJ Publishing Group LTD
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Contents
Background Fabry disease results from deficient α-galactosidase A activity and globotriaosylceramide accumulation causing renal insufficiency, strokes, hypertrophic cardiomyopathy and early demise. We assessed the 10-year outcome of recombinant α-galactosidase A therapy. Methods The outcomes (severe clinical events, renal function, cardiac structur...
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Full title
Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4413801
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4413801
Other Identifiers
ISSN
0022-2593
E-ISSN
1468-6244
DOI
10.1136/jmedgenet-2014-102797
