{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,23]],"date-time":"2026-08-23T16:51:15Z","timestamp":1787503875385,"version":"build-2736575974"},"reference-count":53,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2024,7,4]],"date-time":"2024-07-04T00:00:00Z","timestamp":1720051200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004339","name":"Sanofi","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2024,9]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Fabry Registry data were analyzed among 83 agalsidase beta\u2010treated patients with Fabry disease who switched to migalastat. Outcomes (estimated glomerular filtration rate [eGFR], urine protein\u2010creatinine ratio [UPCR], plasma globotriaosylceramide [GL\u20103], plasma globotriaosylsphingosine [lyso\u2010GL\u20103], interventricular septal wall thickness [IVST], left posterior wall thickness [LPWT], left ventricular mass index [LVMI]) were assessed using linear mixed models to estimate annual change over time in the pre\u2010 and postswitch periods. eGFR decreased throughout both periods (preswitch: \u22120.85\u2009mL\/min\/1.73\u2009m\n                    <jats:sup>2<\/jats:sup>\n                    \/year; postswitch: \u22121.96\u2009mL\/min\/1.73\u2009m\n                    <jats:sup>2<\/jats:sup>\n                    \/year; both\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.0001), with steeper decline postswitch (\n                    <jats:italic>p<\/jats:italic>\n                    <jats:sub>pre\/post<\/jats:sub>\n                    \u2009=\u20090.01) in both classic and late\u2010onset patients. UPCR increased significantly postswitch (\n                    <jats:italic>p<\/jats:italic>\n                    <jats:sub>pre\/post<\/jats:sub>\n                    \u2009=\u20090.003) among classic patients and was stable in both periods among late\u2010onset patients. GL\u20103 trajectories worsened postswitch across phenotypes (\n                    <jats:italic>p<\/jats:italic>\n                    <jats:sub>pre\/post<\/jats:sub>\n                    \u2009=\u20090.0005 classic, 0.02 late\u2010onset). LPWT was stable preswitch (0.07\u2009mm\/year,\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.25) and decreased postswitch (\u22120.51\u2009mm\/year,\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.0005;\n                    <jats:italic>p<\/jats:italic>\n                    <jats:sub>pre\/post<\/jats:sub>\n                    \u2009=\u20090.0009), primarily among late\u2010onset patients. IVST and LVMI slopes varied significantly by phenotype. Among classic patients, IVST and LVMI were stable and decreasing, respectively preswitch and increasing postswitch (\n                    <jats:italic>p<\/jats:italic>\n                    <jats:sub>pre\/post<\/jats:sub>\n                    \u2009=\u20090.02 IVST, 0.01 LVMI). Among late\u2010onset patients, IVST significantly decreased postswitch (\n                    <jats:italic>p<\/jats:italic>\n                    <jats:sub>pre\/post<\/jats:sub>\n                    \u2009=\u20090.0003); LVMI was stable over time (\n                    <jats:italic>p<\/jats:italic>\n                    <jats:sub>pre\/post<\/jats:sub>\n                    \u2009=\u20090.89). Ultimately, eGFR and GL\u20103 trajectories worsened postswitch across phenotypes, while UPCR and cardiac measures worsened among classic and stabilized\/improved among late\u2010onset patients. These findings indicate variability in long\u2010term outcomes after switching from ERT to migalastat, underscoring the importance of careful monitoring.\n                  <\/jats:p>","DOI":"10.1002\/jimd.12773","type":"journal-article","created":{"date-parts":[[2024,7,4]],"date-time":"2024-07-04T03:14:49Z","timestamp":1720062889000},"page":"1080-1095","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Clinical outcomes in patients switching from agalsidase beta to migalastat: A Fabry Registry analysis"],"prefix":"10.1002","volume":"47","author":[{"given":"Antonio","family":"Pisani","sequence":"first","affiliation":[{"name":"Department of Public Health University of Naples Federico II  Naples Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn M.","family":"Wilson","sequence":"additional","affiliation":[{"name":"Navitas Data Sciences  Pottstown Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julie L.","family":"Batista","sequence":"additional","affiliation":[{"name":"Sanofi  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilkka","family":"Kantola","sequence":"additional","affiliation":[{"name":"Division of Medicine Turku University Hospital, Turku University  Turku Finland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alberto","family":"Ortiz","sequence":"additional","affiliation":[{"name":"Jim\u00e9nez D\u00edaz Foundation University Hospital and IIS\u2010Fundaci\u00f3n Jim\u00e9nez D\u00edaz UAM  Madrid Spain"},{"name":"Department of Medicine Universidad Aut\u00f3noma de Madrid  Madrid Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juan","family":"Politei","sequence":"additional","affiliation":[{"name":"Neurology Department Fundaci\u00f3n SPINE  Buenos Aires Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laila","family":"Al\u2010Shaar","sequence":"additional","affiliation":[{"name":"Sanofi  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manish","family":"Maski","sequence":"additional","affiliation":[{"name":"Sanofi  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana","family":"Crespo","sequence":"additional","affiliation":[{"name":"Sanofi  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elvira","family":"Ponce","sequence":"additional","affiliation":[{"name":"Sanofi  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ale\u0161","family":"Linhart","sequence":"additional","affiliation":[{"name":"Department of Cardiovascular Medicine, First Faculty of Medicine Charles University  Prague Czech Republic"},{"name":"General University Hospital  Prague Czech Republic"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,7,4]]},"reference":[{"key":"e_1_2_16_2_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750\u20101172\u20105\u201030"},{"key":"e_1_2_16_3_1","doi-asserted-by":"publisher","DOI":"10.2215\/cjn.09480819"},{"key":"e_1_2_16_4_1","doi-asserted-by":"publisher","DOI":"10.1681\/asn.2016090964"},{"key":"e_1_2_16_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2018.02.014"},{"key":"e_1_2_16_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2020.12.024"},{"key":"e_1_2_16_7_1","doi-asserted-by":"publisher","DOI":"10.3390\/genes15010037"},{"key":"e_1_2_16_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2018.06.004"},{"key":"e_1_2_16_9_1","unstructured":"Genzyme Corporation.Fabrazyme (agalsidase beta) summary of product characteristics. 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