{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,21]],"date-time":"2025-10-21T15:19:13Z","timestamp":1761059953979},"reference-count":15,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2015,6,23]],"date-time":"2015-06-23T00:00:00Z","timestamp":1435017600000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J Med Case Reports"],"published-print":{"date-parts":[[2015,12]]},"DOI":"10.1186\/s13256-015-0617-5","type":"journal-article","created":{"date-parts":[[2015,6,22]],"date-time":"2015-06-22T06:25:06Z","timestamp":1434954306000},"update-policy":"http:\/\/dx.doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series"],"prefix":"10.1186","volume":"9","author":[{"given":"Christine","family":"Serratrice","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Laure","family":"Swiader","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jacques","family":"Serratrice","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2015,6,23]]},"reference":[{"key":"617_CR1","doi-asserted-by":"publisher","first-page":"657","DOI":"10.1016\/S0950-3536(97)80033-9","volume":"10","author":"TM Cox","year":"1997","unstructured":"Cox TM, Schofield JP. Gaucher\u2019s disease: clinical features and natural history. Baillieres Clin Haematol. 1997;10:657\u201389.","journal-title":"Baillieres Clin Haematol."},{"key":"617_CR2","doi-asserted-by":"publisher","first-page":"2835","DOI":"10.1001\/archinte.160.18.2835","volume":"160","author":"J Charrow","year":"2000","unstructured":"Charrow J, Andersson HC, Kaplan P, Kolodny EH, Mistry P, Pastores G, et al. The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease. Arch Intern Med. 2000;160:2835\u201343.","journal-title":"Arch Intern Med."},{"key":"617_CR3","first-page":"407","volume":"3","author":"D Elstein","year":"2009","unstructured":"Elstein D, Zimran A. Review of the safety and efficacy of imiglucerase treatment of Gaucher disease. Biologics. 2009;3:407\u201317.","journal-title":"Biologics."},{"key":"617_CR4","doi-asserted-by":"publisher","first-page":"1182","DOI":"10.1542\/peds.2007-2144","volume":"122","author":"H Andersson","year":"2008","unstructured":"Andersson H, Kaplan P, Kacena K, Yee J. Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1. Pediatrics. 2008;122:1182\u201390.","journal-title":"Pediatrics."},{"key":"617_CR5","doi-asserted-by":"publisher","first-page":"1481","DOI":"10.1016\/S0140-6736(00)02161-9","volume":"355","author":"T Cox","year":"2000","unstructured":"Cox T, Lachmann R, Hollak C, Aerts J, van Weely S, Hrebicek M, et al. Novel oral treatment of Gaucher\u2019s disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis. Lancet. 2000;355:1481\u20135.","journal-title":"Lancet."},{"key":"617_CR6","doi-asserted-by":"publisher","first-page":"127","DOI":"10.1006\/bcmd.2002.0497","volume":"28","author":"R Heitner","year":"2002","unstructured":"Heitner R, Elstein D, Aerts J, Weely S, Zimran A. Low-dose N-butyldeoxynojirimycin (OGT 918) for type I Gaucher disease. Blood Cells Mol Dis. 2002;28:127\u201333.","journal-title":"Blood Cells Mol Dis."},{"key":"617_CR7","doi-asserted-by":"publisher","first-page":"757","DOI":"10.1023\/B:BOLI.0000045756.54006.17","volume":"27","author":"D Elstein","year":"2004","unstructured":"Elstein D, Hollak C, Aerts JM, van Weely S, Maas M, Cox TM, et al. Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease. J Inherit Metab Dis. 2004;27:757\u201366.","journal-title":"J Inherit Metab Dis."},{"key":"617_CR8","doi-asserted-by":"publisher","first-page":"1645","DOI":"10.1016\/j.clinthera.2007.08.006","volume":"29","author":"GM Pastores","year":"2007","unstructured":"Pastores GM, Elstein D, Hrebicek M, Zimran A. Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies. Clin Ther. 2007;29:1645\u201354.","journal-title":"Clin Ther."},{"key":"617_CR9","doi-asserted-by":"publisher","first-page":"41","DOI":"10.1016\/j.bcmd.2009.09.006","volume":"44","author":"CE Hollak","year":"2010","unstructured":"Hollak CE, Vom Dahl S, Aerts JM, Belmatoug N, Bembi B, Cohen Y, et al. Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease. Blood Cells Mol Dis. 2010;44:41\u20137.","journal-title":"Blood Cells Mol Dis."},{"key":"617_CR10","doi-asserted-by":"publisher","first-page":"1771","DOI":"10.3324\/haematol.2009.008078","volume":"94","author":"P Giraldo","year":"2009","unstructured":"Giraldo P, Alfonso P, Atutxa K, Fernandez-Galan MA, Barez A, Franco R, et al. Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project. Haematologica. 2009;94:1771\u20135.","journal-title":"Haematologica."},{"key":"617_CR11","doi-asserted-by":"publisher","first-page":"102","DOI":"10.1186\/1750-1172-7-102","volume":"7","author":"TM Cox","year":"2012","unstructured":"Cox TM, Amato D, Hollak CE, Luzy C, Silkey M, Giorgino R, et al. Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study. Orphanet J Rare Dis. 2012;7:102.","journal-title":"Orphanet J Rare Dis."},{"key":"617_CR12","doi-asserted-by":"publisher","first-page":"116","DOI":"10.1016\/j.bcmd.2013.04.005","volume":"51","author":"DJ Kuter","year":"2013","unstructured":"Kuter DJ, Mehta A, Hollak CE, Giraldo P, Hughes D, Belmatoug N, et al. Miglustat therapy in type 1 Gaucher disease: clinical and safety outcomes in a multicenter retrospective cohort study. Blood Cells Mol Dis. 2013;51:116\u201324.","journal-title":"Blood Cells Mol Dis."},{"key":"617_CR13","doi-asserted-by":"publisher","first-page":"2909","DOI":"10.1093\/brain\/awq198","volume":"133","author":"M Biegstraaten","year":"2010","unstructured":"Biegstraaten M, Mengel E, Marodi L, Petakov M, Niederau C, Giraldo P, et al. Peripheral neuropathy in adult type 1 Gaucher disease: a 2-year prospective observational study. Brain. 2010;133:2909\u201319.","journal-title":"Brain."},{"key":"617_CR14","doi-asserted-by":"publisher","first-page":"991","DOI":"10.1007\/s10545-011-9368-7","volume":"34","author":"N Belmatoug","year":"2011","unstructured":"Belmatoug N, Burlina A, Giraldo P, Hendriksz CJ, Kuter DJ, Mengel E, et al. Gastrointestinal disturbances and their management in miglustat-treated patients. J Inherit Metab Dis. 2011;34:991\u20131001.","journal-title":"J Inherit Metab Dis."},{"key":"617_CR15","unstructured":"Miglustat (Zavesca) Summary of Product Characteristics (EudraPharm). [\n                    http:\/\/www.eudrapharm.eu\/eudrapharm\/productDetailsAction.do\n                    \n                  ]"}],"container-title":["Journal of Medical Case Reports"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1186\/s13256-015-0617-5.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/article\/10.1186\/s13256-015-0617-5\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1186\/s13256-015-0617-5.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1186\/s13256-015-0617-5","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,3,26]],"date-time":"2019-03-26T18:52:44Z","timestamp":1553626364000},"score":1,"resource":{"primary":{"URL":"https:\/\/jmedicalcasereports.biomedcentral.com\/articles\/10.1186\/s13256-015-0617-5"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2015,6,23]]},"references-count":15,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2015,12]]}},"alternative-id":["617"],"URL":"https:\/\/doi.org\/10.1186\/s13256-015-0617-5","relation":{},"ISSN":["1752-1947"],"issn-type":[{"value":"1752-1947","type":"electronic"}],"subject":[],"published":{"date-parts":[[2015,6,23]]},"assertion":[{"value":"28 November 2014","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"20 May 2015","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"23 June 2015","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}}],"article-number":"146"}}