{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T20:24:49Z","timestamp":1793391889914,"version":"4.5.0"},"reference-count":71,"publisher":"Oxford University Press (OUP)","issue":"Supplement_2","license":[{"start":{"date-parts":[[2021,6,1]],"date-time":"2021-06-01T00:00:00Z","timestamp":1622505600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"funder":[{"name":"Amicus Therapeutics UK Limited"},{"name":"Boehringer Ingelheim RCV GmbH & Co KG"},{"DOI":"10.13039\/501100004948","name":"Astellas Pharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004948","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Vifor Pharma Osterreich GmbH"},{"name":"European Union's Horizon 2020 research and innovation programme","award":["848011"],"award-info":[{"award-number":["848011"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2021,6,22]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Fabry disease (FD) is a rare X-linked lysosomal storage disorder caused by mutations in the \u03b1-galactosidase A (GLA) gene, leading to a deficiency in \u03b1-galactosidase A. The lysosomal accumulation of glycosphingolipids, primarily globotriaosylceramide (Gb3) and its deacylated form, globotriaosylsphingosine (lyso-Gb3), results in progressive renal failure, cardiomyopathy associated with cardiac arrhythmia and recurrent cerebrovascular events, significantly limiting life expectancy in affected patients. In male patients, a definitive diagnosis of FD involves demonstrating a GLA deficiency in leucocytes. In females, because of the potential high residual enzymatic activity, the diagnostic gold standard requires molecular genetic analyses. The current treatment options for FD include recombinant enzyme replacement therapies (ERTs) with intravenous agalsidase-\u03b1 (0.2\u2009mg\/kg body weight) or agalsidase-\u03b2 (1\u2009mg\/kg body weight) every 2\u2009weeks as well as an oral pharmacological chaperone (migalastat 123\u2009mg every other day) that selectively and reversibly binds to the active sites of amenable mutant forms of the GLA enzyme. These therapies facilitate cellular Gb3 clearance and an overall improvement of disease burden. However, ERT can lead to infusion-associated reactions, as well as the formation of neutralizing anti-drug antibodies in \u223c40% of all ERT-treated males, leading to an attenuation of therapy efficacy. This article reviews the clinical presentation, diagnosis and interdisciplinary clinical management of FD and discusses the therapeutic options, with a special focus on precision medicine, accounting for individual variability in genetic mutations, Gb3 and lyso-Gb3 levels, allowing physicians to predict more accurately which prevention and treatment strategy is best for which patient.<\/jats:p>","DOI":"10.1093\/ndt\/gfab038","type":"journal-article","created":{"date-parts":[[2021,2,10]],"date-time":"2021-02-10T03:08:26Z","timestamp":1612926506000},"page":"14-23","source":"Crossref","is-referenced-by-count":35,"title":["Precision medicine in Fabry disease"],"prefix":"10.1093","volume":"36","author":[{"given":"Malte","family":"Lenders","sequence":"first","affiliation":[{"name":"Internal Medicine D, Department of Nephrology, Hypertension and Rheumatology, Interdisciplinary Fabry Center M\u00fcnster, University Hospital M\u00fcnster, M\u00fcnster, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eva","family":"Brand","sequence":"additional","affiliation":[{"name":"Internal Medicine D, Department of Nephrology, Hypertension and Rheumatology, Interdisciplinary Fabry Center M\u00fcnster, University Hospital M\u00fcnster, M\u00fcnster, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2021,6,18]]},"reference":[{"key":"2021070723095604500_gfab038-B1","doi-asserted-by":"crossref","first-page":"1427","DOI":"10.1016\/S0140-6736(08)61589-5","article-title":"Fabry's disease","volume":"372","author":"Zarate","year":"2008","journal-title":"Lancet"},{"key":"2021070723095604500_gfab038-B2","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1086\/504601","article-title":"High incidence of later-onset Fabry disease revealed by newborn screening","volume":"79","author":"Spada","year":"2006","journal-title":"Am J Hum 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