{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T19:53:41Z","timestamp":1793044421160,"version":"4.5.0"},"reference-count":60,"publisher":"MDPI AG","issue":"8","license":[{"start":{"date-parts":[[2023,8,17]],"date-time":"2023-08-17T00:00:00Z","timestamp":1692230400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Medicina"],"abstract":"<jats:p>Fabry disease is an X-linked inherited lysosomal storage disorder with a deficiency of \u03b1-galactosidase A activity, which results in the intracellular accumulation of globotriaosylceramide (Gb3) and related glycosphingolipids in various organs. Fabry nephropathy is one of the major complications of Fabry disease, and kidney damage is often related to cardiovascular disease and mortality. The treatment of Fabry nephropathy thus helps prolong life expectancy. Two treatment options for Fabry nephropathy and cardiopathy are now commercially available: enzyme replacement therapy (agalsidase \u03b1 agalsidase \u03b2, and a biosimilar of agalsidase \u03b2) and pharmacological chaperone therapy (migalastat). In this review, we summarize the efficacy of these treatment options for Fabry nephropathy with respect to renal function, proteinuria, and renal pathological findings. We also describe the importance of adjunctive therapy for Fabry nephropathy.<\/jats:p>","DOI":"10.3390\/medicina59081478","type":"journal-article","created":{"date-parts":[[2023,8,17]],"date-time":"2023-08-17T10:15:48Z","timestamp":1692267348000},"page":"1478","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":12,"title":["Treatment of Fabry Nephropathy: A Literature Review"],"prefix":"10.3390","volume":"59","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9648-5908","authenticated-orcid":false,"given":"Homare","family":"Shimohata","sequence":"first","affiliation":[{"name":"Department of Nephrology, Tokyo Medical University Ibaraki Medical Center, Ami 300-0395, Ibaraki, Japan"},{"name":"Tsuchiura Beryl Clinic, Tsuchiura 300-0062, Ibaraki, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marina","family":"Yamashita","sequence":"additional","affiliation":[{"name":"Department of Nephrology, Tokyo Medical University Ibaraki Medical Center, Ami 300-0395, Ibaraki, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kota","family":"Yamada","sequence":"additional","affiliation":[{"name":"Tsuchiura Beryl Clinic, Tsuchiura 300-0062, Ibaraki, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2016-8276","authenticated-orcid":false,"given":"Kouichi","family":"Hirayama","sequence":"additional","affiliation":[{"name":"Department of Nephrology, Tokyo Medical University Ibaraki Medical Center, Ami 300-0395, Ibaraki, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masaki","family":"Kobayashi","sequence":"additional","affiliation":[{"name":"Ohba Medical Clinic, Mito 310-0841, Ibaraki, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2023,8,17]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"1163","DOI":"10.1056\/NEJM196705252762101","article-title":"Enzyme defect in Fabry\u2019s disease. 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