Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal,...
Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes
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Publisher
England: BMJ Publishing Group Ltd
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Language
English
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England: BMJ Publishing Group Ltd
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BackgroundFabry disease is a rare, multisystemic disorder caused by GLA gene variants that lead to alpha galactosidase A deficiency, resulting in accumulation of glycosphingolipids and cellular dysfunction. Fabry-associated clinical events (FACEs) cause significant morbidity and mortality, yet the long-term effect of Fabry therapies on FACE inciden...
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Full title
Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_10359570
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_10359570
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ISSN
0022-2593
E-ISSN
1468-6244
DOI
10.1136/jmg-2022-108669
