{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T01:20:09Z","timestamp":1788830409409,"version":"build-2803163510"},"reference-count":11,"publisher":"SAGE Publications","issue":"4","license":[{"start":{"date-parts":[[2007,7,1]],"date-time":"2007-07-01T00:00:00Z","timestamp":1183248000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J Int Med Res"],"published-print":{"date-parts":[[2007,7]]},"abstract":"<jats:p>Fabry's disease, a disorder affecting the gene for the lysosomal enzyme \u03b1-galactosidase A (\u03b1-GAL A), can cause accumulation of globotriaosylceramide (GL-3) in the vascular endothelial cells. Symptoms include pain, angiokeratoma, corneal clouding, and damage to the heart and kidneys. Human recombinant \u03b1-GAL A for use as an enzyme replacement therapy was launched in Japan in April 2004. Eleven ambulatory patients with Fabry's disease were given replacement \u03b1-GAL A therapy. Three patients died due to factors associated with Fabry's disease. The enzyme replacement therapies in the remaining eight patients continued safely without any notable adverse events. The following were observed: a lowering of the plasma levels of GL-3 in seven cases, an improvement in the daily activities in six cases, and a reduction in corneal clouding in three cases. Although careful observation is necessary, these results suggest that replacement \u03b1-GAL A therapy may be a safe and effective treatment of Fabry's disease.<\/jats:p>","DOI":"10.1177\/147323000703500418","type":"journal-article","created":{"date-parts":[[2013,2,27]],"date-time":"2013-02-27T14:22:43Z","timestamp":1361974963000},"page":"574-581","source":"Crossref","is-referenced-by-count":12,"title":["Enzyme Replacement Therapy in Patients with Fabry's Disease"],"prefix":"10.1177","volume":"35","author":[{"given":"K","family":"Tsuboi","sequence":"first","affiliation":[{"name":"Department of Haematology, Nagoya Central Hospital, Nagoya, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2007,7,1]]},"reference":[{"key":"bibr1-147323000703500418","first-page":"2741","volume-title":"Metabolic and Molecular Bases of Inherited Disease","author":"Desnick RJ","year":"1995"},{"key":"bibr2-147323000703500418","doi-asserted-by":"publisher","DOI":"10.1086\/318809"},{"key":"bibr3-147323000703500418","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200107053450102"},{"key":"bibr4-147323000703500418","doi-asserted-by":"publisher","DOI":"10.1111\/j.1742-1241.2006.01237.x"},{"key":"bibr5-147323000703500418","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-005-0575-y"},{"key":"bibr6-147323000703500418","doi-asserted-by":"publisher","DOI":"10.1016\/0003-9993(93)90153-2"},{"key":"bibr7-147323000703500418","doi-asserted-by":"publisher","DOI":"10.1016\/0003-9993(94)90386-7"},{"key":"bibr8-147323000703500418","doi-asserted-by":"publisher","DOI":"10.1086\/422366"},{"key":"bibr9-147323000703500418","doi-asserted-by":"publisher","DOI":"10.1046\/j.1523-1755.2002.00675.x"},{"key":"bibr10-147323000703500418","first-page":"21","volume-title":"Fabry's Disease: Recent Findings, Basic and Clinical Research","author":"Sakuraba H","year":"2004"},{"key":"bibr11-147323000703500418","first-page":"3","volume-title":"Fabry's Disease: Recent Findings, Basic and Clinical Research","author":"Eto Y","year":"2004"}],"container-title":["Journal of International Medical Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/147323000703500418","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/147323000703500418","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T10:41:03Z","timestamp":1777459263000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/147323000703500418"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2007,7]]},"references-count":11,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2007,7]]}},"alternative-id":["10.1177\/147323000703500418"],"URL":"https:\/\/doi.org\/10.1177\/147323000703500418","relation":{},"ISSN":["0300-0605","1473-2300"],"issn-type":[{"value":"0300-0605","type":"print"},{"value":"1473-2300","type":"electronic"}],"subject":[],"published":{"date-parts":[[2007,7]]}}}