{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,22]],"date-time":"2025-10-22T18:13:16Z","timestamp":1761156796930},"reference-count":22,"publisher":"Springer Science and Business Media LLC","issue":"2","license":[{"start":{"date-parts":[[2019,12,30]],"date-time":"2019-12-30T00:00:00Z","timestamp":1577664000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2019,12,30]],"date-time":"2019-12-30T00:00:00Z","timestamp":1577664000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Clin Exp Nephrol"],"published-print":{"date-parts":[[2020,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec>\n                <jats:title>Background<\/jats:title>\n                <jats:p>Fabry disease is a progressive X-linked lysosomal disorder. In this subgroup analysis of the global phase III ATTRACT study, the efficacy and safety of oral migalastat, a pharmacologic chaperone, were investigated in Japanese patients with Fabry disease.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>Patients were randomly assigned to receive migalastat (150\u00a0mg every other day) or to continue biweekly enzyme replacement therapy infusions (ERT; agalsidase alfa 0.2\u00a0mg\/kg or agalsidase beta 1.0\u00a0mg\/kg) for 18\u00a0months followed by a 12-month open-label extension during which all patients received migalastat. End points included glomerular filtration rate (estimated and measured), left ventricular mass index (LVMi), composite clinical outcomes, leukocyte alpha-galactosidase A activity, plasma globotriaosylsphingosine (lyso-Gb<jats:sub>3<\/jats:sub>), and safety.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>Data from 7 Japanese patients (migalastat, 5; ERT, 2), mean age 55\u00a0years, with high disease burden, were analyzed. All patients in the migalastat group completed the open-label comparison and extension periods. At 18\u00a0months, efficacy in the Japanese patient population was similar to that in the overall ATTRACT population. Migalastat treatment increased leukocyte alpha-galactosidase A activity, stabilized renal function, and decreased LVMi. Plasma lyso-Gb<jats:sub>3<\/jats:sub> levels remained low and stable. Additionally, the long-term extension study showed that efficacy of migalastat was maintained for up to 48\u00a0months. Migalastat was safe and well tolerated in the Japanese patients, as in the overall ATTRACT population.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusion<\/jats:title>\n                <jats:p>Migalastat can be used to treat Japanese patients with Fabry disease with <jats:italic>GLA<\/jats:italic> mutations amenable to migalastat according to the dosage and administration approved in other countries.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Trial registration numbers<\/jats:title>\n                <jats:p>ClinicalTrials.gov, NCT01218659 and NCT02194985.<\/jats:p>\n              <\/jats:sec>","DOI":"10.1007\/s10157-019-01810-w","type":"journal-article","created":{"date-parts":[[2019,12,30]],"date-time":"2019-12-30T08:02:21Z","timestamp":1577692941000},"page":"157-166","update-policy":"http:\/\/dx.doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study"],"prefix":"10.1007","volume":"24","author":[{"given":"Ichiei","family":"Narita","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Toya","family":"Ohashi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Norio","family":"Sakai","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Takashi","family":"Hamazaki","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nina","family":"Skuban","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jeffrey P.","family":"Castelli","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hjalmar","family":"Lagast","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jay A.","family":"Barth","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2019,12,30]]},"reference":[{"issue":"2","key":"1810_CR1","doi-asserted-by":"publisher","first-page":"285","DOI":"10.1042\/BJ20070479","volume":"406","author":"S Ishii","year":"2007","unstructured":"Ishii S, Chang HH, Kawasaki K, et al. 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J Inherit Metab Dis. 2007;30(2):184\u201392.","journal-title":"J Inherit Metab Dis"}],"container-title":["Clinical and Experimental Nephrology"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s10157-019-01810-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/article\/10.1007\/s10157-019-01810-w\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s10157-019-01810-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,12,29]],"date-time":"2020-12-29T00:05:25Z","timestamp":1609200325000},"score":1,"resource":{"primary":{"URL":"http:\/\/link.springer.com\/10.1007\/s10157-019-01810-w"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,12,30]]},"references-count":22,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2020,2]]}},"alternative-id":["1810"],"URL":"https:\/\/doi.org\/10.1007\/s10157-019-01810-w","relation":{},"ISSN":["1342-1751","1437-7799"],"issn-type":[{"value":"1342-1751","type":"print"},{"value":"1437-7799","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,12,30]]},"assertion":[{"value":"19 July 2019","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"28 October 2019","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"30 December 2019","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Compliance with ethical standards"}},{"value":"Employment: Nina Skuban, Jeffrey P. Castelli, Hjalmar Lagast, and Jay A. Barth (Amicus Therapeutics Inc.); stock ownership or options: Nina Skuban, Jeffrey P. Castelli, Hjalmar Lagast, and Jay A. Barth (Amicus Therapeutics Inc.); research grant: Toyo Ohashi (ARVOBIO Inc., Dainippon Sumitomo Pharma, Sanofi Genzyme, Amicus Therapeutics Inc.) and Noroi Sakai (Sanofi Genzyme, Dainippon Sumitomo Pharma); Honoraria: Toyo Ohashi (Dainippon Sumitomo Pharma) and Norio Sakai (Sanofi Genzyme, Dainippon Sumitomo Pharma). All other authors have declared that no conflict of interest exists.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}},{"value":"All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and\/or national research committee (IRB approval number for AT1001-012 and AT1001-042: The Jikei University School of Medicine: 23-25(2845) and 26-07(2935); Osaka City University Graduate School of Medicine: 1688 and 1827; Osaka University Graduate School of Medicine: 124002 and 146062; Niigata University Graduate School of Medical and Dental Sciences: CH23-017 and CH26-013) at which the studies were conducted according to International Conference on Harmonisation\/Good clinical Practice (ICH\/GCP) and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}},{"value":"Informed consent was obtained from all participants included in the present study.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Informed consent"}}]}}