{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,22]],"date-time":"2025-10-22T02:57:46Z","timestamp":1761101866251},"reference-count":87,"publisher":"Informa Healthcare","issue":"16","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Expert Opinion on Pharmacotherapy"],"published-print":{"date-parts":[[2009,11]]},"DOI":"10.1517\/14656560903270520","type":"journal-article","created":{"date-parts":[[2009,9,11]],"date-time":"2009-09-11T05:05:19Z","timestamp":1252645519000},"page":"2641-2652","source":"Crossref","is-referenced-by-count":22,"title":["Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose \u2013 response relationships"],"prefix":"10.1517","volume":"10","author":[{"given":"Carla EM","family":"Hollak","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maaike","family":"de Fost","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Laura","family":"van Dussen","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stephan","family":"vom Dahl","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Johannes MFG","family":"Aerts","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"3197","published-online":{"date-parts":[[2009,9,10]]},"reference":[{"key":"CIT0003","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1016\/0006-291X(65)90743-6","volume":"18","author":"Brady RO","year":"1965","journal-title":"Biochem Biophys Res Commun"},{"key":"CIT0004","doi-asserted-by":"crossref","first-page":"17C","DOI":"10.1042\/bj0970017C","volume":"97","author":"Patrick AD","year":"1955","journal-title":"Biochem J"},{"key":"CIT0005","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1016\/j.ymgme.2004.08.015","volume":"83","author":"Sidransky E","year":"2004","journal-title":"Mol Genet Metab"},{"key":"CIT0006","doi-asserted-by":"publisher","DOI":"10.1016\/S0950-3536(97)80033-9"},{"key":"CIT0007","unstructured":"Beutler E, Grabowski GA. Gaucher disease. In: Scriver CR, Beaudet AL, Sly WS, Valle D, editors, The metabolic and molecular bases of inherited disease, 8th edition. New York: McGraw \u2013 Hill; 2001. p. 3635-68"},{"key":"CIT0008","first-page":"85","volume":"52","author":"Beutler E","year":"1993","journal-title":"Am J Hum Genet"},{"key":"CIT0009","first-page":"855","volume":"49","author":"Zimran A","year":"1991","journal-title":"Am J Hum Genet"},{"key":"CIT0010","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1001\/jama.281.3.249","volume":"281","author":"Meikle PJ","year":"1999","journal-title":"JAMA"},{"key":"CIT0011","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1089\/gte.1997.1.5","volume":"1","author":"Grabowski GA","year":"1997","journal-title":"Genet Test"},{"key":"CIT0012","doi-asserted-by":"crossref","first-page":"155","DOI":"10.1016\/0009-8981(86)90231-7","volume":"158","author":"Aerts JM","year":"1986","journal-title":"Clin Chim Acta"},{"key":"CIT0013","doi-asserted-by":"crossref","first-page":"569","DOI":"10.1093\/clinchem\/28.4.569","volume":"28","author":"Daniels LB","year":"1982","journal-title":"Clin Chem"},{"key":"CIT0014","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1007\/s00277-008-0441-8","volume":"87","author":"de Fost M","year":"2008","journal-title":"Ann Hematol"},{"key":"CIT0015","doi-asserted-by":"crossref","first-page":"1215","DOI":"10.1172\/JCI0214530","volume":"109","author":"Mizukami H","year":"2002","journal-title":"J Clin Invest"},{"key":"CIT0016","first-page":"237","volume":"93","author":"Lachmann RH","year":"2000","journal-title":"features QJM"},{"key":"CIT0017","doi-asserted-by":"crossref","first-page":"748","DOI":"10.2106\/00004623-200105000-00016","volume":"83","author":"Mankin HJ","year":"2001","journal-title":"J Bone Joint Surg Am"},{"key":"CIT0018","doi-asserted-by":"crossref","first-page":"297","DOI":"10.1097\/00003072-200004000-00017","volume":"25","author":"Mansberg R","year":"2000","journal-title":"Clin Nucl Med"},{"key":"CIT0019","doi-asserted-by":"publisher","DOI":"10.1016\/S1096-7192(02)00122-1"},{"key":"CIT0020","doi-asserted-by":"crossref","first-page":"783","DOI":"10.1161\/01.CIR.35.4.783","volume":"35","author":"Roberts WC","year":"1967","journal-title":"Circulation"},{"key":"CIT0021","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1016\/0002-9343(78)90832-X","volume":"65","author":"Smith RL","year":"1978","journal-title":"Am J Med"},{"key":"CIT0022","doi-asserted-by":"publisher","DOI":"10.1016\/j.bcmd.2005.08.004"},{"key":"CIT0023","doi-asserted-by":"publisher","DOI":"10.1016\/j.bcmd.2005.03.004"},{"key":"CIT0024","doi-asserted-by":"publisher","DOI":"10.1093\/qjmed\/89.9.691"},{"key":"CIT0025","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1006\/mgme.2001.3201","volume":"73","author":"Tayebi N","year":"2001","journal-title":"Mol Genet Metab"},{"key":"CIT0026","doi-asserted-by":"publisher","DOI":"10.1517\/14656566.9.11.1987"},{"key":"CIT0027","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1097\/00019052-199904000-00007","volume":"12","author":"Krivit W","year":"1999","journal-title":"Curr Opin Neurol"},{"key":"CIT0028","doi-asserted-by":"publisher","DOI":"10.1126\/science.162.3853.570"},{"key":"CIT0029","doi-asserted-by":"publisher","DOI":"10.1016\/0006-291X(78)91456-0"},{"key":"CIT0030","first-page":"547","volume":"23","author":"Sly WS","year":"1978","journal-title":"Prog Clin Biol Res"},{"key":"CIT0031","doi-asserted-by":"crossref","first-page":"1464","DOI":"10.1056\/NEJM199105233242104","volume":"324","author":"Barton NW","year":"1991","journal-title":"N Engl J Med"},{"key":"CIT0032","doi-asserted-by":"crossref","first-page":"33","DOI":"10.7326\/0003-4819-122-1-199501010-00005","volume":"122","author":"Grabowski GA","year":"1995","journal-title":"Ann Intern Med"},{"key":"CIT0033","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(00)02161-9"},{"key":"CIT0034","doi-asserted-by":"crossref","first-page":"2643","DOI":"10.1002\/cbic.200800249","volume":"9","author":"Kornhaber GJ","year":"2008","journal-title":"Chembiochem"},{"key":"CIT0035","doi-asserted-by":"crossref","first-page":"13819","DOI":"10.1073\/pnas.0606016103","volume":"103","author":"Enquist IB","year":"2006","journal-title":"Proc Natl Acad Sci USA"},{"key":"CIT0037","doi-asserted-by":"crossref","first-page":"2093","DOI":"10.1016\/S0002-9440(10)63566-3","volume":"163","author":"Xu YH","year":"2003","journal-title":"Am J Pathol"},{"key":"CIT0038","doi-asserted-by":"crossref","first-page":"467","DOI":"10.1093\/glycob\/cwm008","volume":"17","author":"Van Patten SM","year":"2007","journal-title":"Glycobiology"},{"key":"CIT0039","doi-asserted-by":"publisher","DOI":"10.1172\/JCI116409"},{"key":"CIT0040","doi-asserted-by":"crossref","first-page":"2807","DOI":"10.1182\/blood.V93.9.2807","volume":"93","author":"Friedman B","year":"1999","journal-title":"Blood"},{"key":"CIT0041","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(96)04451-0"},{"key":"CIT0042","doi-asserted-by":"crossref","first-page":"2081","DOI":"10.1182\/blood.V93.6.2081.406k07_2081_2088","volume":"93","author":"Rosenberg M","year":"1999","journal-title":"Blood"},{"key":"CIT0043","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1182\/blood.V90.1.43","volume":"90","author":"Ponce E","year":"1997","journal-title":"Blood"},{"key":"CIT0044","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/S1079-9796(03)00012-3","volume":"30","author":"Zhao H","year":"2003","journal-title":"Blood Cells Mol Dis"},{"key":"CIT0045","doi-asserted-by":"crossref","first-page":"330","DOI":"10.12968\/bjon.2006.15.6.20681","volume":"15","author":"Milligan A","year":"2006","journal-title":"Br J Nurs"},{"key":"CIT0046","doi-asserted-by":"crossref","first-page":"1107","DOI":"10.1182\/blood.V82.4.1107.1107","volume":"82","author":"Zimran A","year":"1993","journal-title":"Blood"},{"key":"CIT0047","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.87.5.1913"},{"key":"CIT0048","doi-asserted-by":"crossref","first-page":"1632","DOI":"10.1056\/NEJM199212033272304","volume":"327","author":"Figueroa ML","year":"1992","journal-title":"N Engl J Med"},{"key":"CIT0049","doi-asserted-by":"crossref","first-page":"408","DOI":"10.1182\/blood.V82.2.408.408","volume":"82","author":"Pastores GM","year":"1993","journal-title":"Blood"},{"key":"CIT0050","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1016\/0002-9343(94)90042-6","volume":"97","author":"Zimran A","year":"1994","journal-title":"Am J Med"},{"key":"CIT0051","doi-asserted-by":"crossref","first-page":"1474","DOI":"10.1016\/S0140-6736(95)91037-9","volume":"345","author":"Hollak CE","year":"1995","journal-title":"Lancet"},{"key":"CIT0052","doi-asserted-by":"crossref","first-page":"1182","DOI":"10.1542\/peds.2007-2144","volume":"122","author":"Andersson H","year":"2008","journal-title":"Pediatrics"},{"key":"CIT0053","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1016\/j.jpeds.2003.10.067","volume":"144","author":"Charrow J","year":"2004","journal-title":"J Pediatr"},{"key":"CIT0054","first-page":"541","volume":"83","author":"Mistry PK","year":"1992","journal-title":"QJM"},{"key":"CIT0055","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1016\/S0002-9343(02)01150-6","volume":"113","author":"Weinreb NJ","year":"2002","journal-title":"Am J Med"},{"key":"CIT0056","doi-asserted-by":"crossref","first-page":"783","DOI":"10.1111\/j.1365-2141.1993.tb03224.x","volume":"85","author":"Zimran A","year":"1993","journal-title":"Br J Haematol"},{"key":"CIT0057","doi-asserted-by":"crossref","first-page":"1479","DOI":"10.1016\/S0140-6736(95)91038-7","volume":"345","author":"Zimran A","year":"1995","journal-title":"Lancet"},{"key":"CIT0058","doi-asserted-by":"crossref","first-page":"961","DOI":"10.2214\/ajr.179.4.1790961","volume":"179","author":"Maas M","year":"2002","journal-title":"AJR Am J Roentgenol"},{"key":"CIT0059","doi-asserted-by":"publisher","DOI":"10.1185\/030079906X104623"},{"key":"CIT0060","doi-asserted-by":"publisher","DOI":"10.1080\/08035320510028094"},{"key":"CIT0061","doi-asserted-by":"crossref","first-page":"1288","DOI":"10.1172\/JCI117084","volume":"93","author":"Hollak CE","year":"1994","journal-title":"J Clin Invest"},{"key":"CIT0062","doi-asserted-by":"crossref","first-page":"26252","DOI":"10.1074\/jbc.270.44.26252","volume":"270","author":"Boot RG","year":"1995","journal-title":"J Biol Chem"},{"issue":"2","key":"CIT0063","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1023\/A:1012492429191","volume":"24","author":"Hollak CE","year":"2001","journal-title":"J Inherit Metab Dis"},{"key":"CIT0064","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-05-1612"},{"key":"CIT0065","doi-asserted-by":"crossref","first-page":"1969","DOI":"10.1182\/blood.V96.5.1969","volume":"96","author":"Moran MT","year":"2000","journal-title":"Blood"},{"key":"CIT0066","doi-asserted-by":"crossref","first-page":"788","DOI":"10.1016\/j.bbadis.2007.04.002","volume":"1772","author":"van Breemen MJ","year":"2007","journal-title":"Biochim Biophys Acta"},{"key":"CIT0067","doi-asserted-by":"crossref","first-page":"751","DOI":"10.1016\/S0950-3536(97)80038-8","volume":"10","author":"Beutler E","year":"1997","journal-title":"Baillieres Clin Haematol"},{"key":"CIT0068","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1006\/bcmd.2000.0310","volume":"26","author":"Altarescu G","year":"2000","journal-title":"Blood Cells Mol Dis"},{"key":"CIT0069","doi-asserted-by":"crossref","first-page":"830","DOI":"10.1182\/blood-2005-12-5072","volume":"108","author":"de Fost M","year":"2006","journal-title":"Blood"},{"key":"CIT0070","doi-asserted-by":"publisher","DOI":"10.1359\/jbmr.061004"},{"key":"CIT0071","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1097\/GIM.0b013e31818e2c19","volume":"11","author":"Grabowski GA","year":"2009","journal-title":"Genet Med"},{"key":"CIT0072","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1097\/GIM.0b013e3181928f6a","volume":"11","author":"Sidransky E","year":"2009","journal-title":"Genet Med"},{"key":"CIT0073","doi-asserted-by":"publisher","DOI":"10.1053\/j.seminhematol.2004.07.009"},{"key":"CIT0074","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1023\/A:1008814105603","volume":"7","author":"Damiano AM","year":"1998","journal-title":"Qual Life Res"},{"key":"CIT0075","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-004-0794-y"},{"key":"CIT0076","doi-asserted-by":"publisher","DOI":"10.1023\/A:1008859420641"},{"key":"CIT0077","doi-asserted-by":"crossref","first-page":"576","DOI":"10.1111\/j.1399-0004.2007.00811.x","volume":"71","author":"Weinreb N","year":"2007","journal-title":"Clin Genet"},{"key":"CIT0078","doi-asserted-by":"crossref","first-page":"890","DOI":"10.1002\/ajh.21280","volume":"83","author":"Weinreb N","year":"2008","journal-title":"Am J Hematol"},{"key":"CIT0079","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1097\/01.GIM.0000153660.88672.3C","volume":"7","author":"Andersson HC","year":"2005","journal-title":"Genet Med"},{"key":"CIT0080","doi-asserted-by":"crossref","first-page":"319","DOI":"10.1007\/s10545-008-0779-z","volume":"31","author":"Cox TM","year":"2008","journal-title":"J Inherit Metab Dis"},{"key":"CIT0081","doi-asserted-by":"crossref","first-page":"1119","DOI":"10.3324\/haematol.12651","volume":"93","author":"de Fost M","year":"2008","journal-title":"Haematologica"},{"key":"CIT0082","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1016\/j.ymgme.2008.11.158","volume":"96","author":"Brunel-Guitton C","year":"2009","journal-title":"Mol Genet Metab"},{"key":"CIT0083","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1007\/PL00008411","volume":"160","author":"Ida H","year":"2001","journal-title":"Eur J Pediatr"},{"key":"CIT0084","doi-asserted-by":"crossref","first-page":"215","DOI":"10.3324\/haematol.10635","volume":"92","author":"de Fost M","year":"2007","journal-title":"Haematologica"},{"key":"CIT0085","doi-asserted-by":"crossref","first-page":"164","DOI":"10.1016\/j.ymgme.2008.12.015","volume":"96","author":"Kishnani PS","year":"2009","journal-title":"Mol Genet Metab"},{"key":"CIT0086","doi-asserted-by":"crossref","first-page":"488","DOI":"10.1046\/j.1365-2141.2000.02177.x","volume":"110","author":"Elstein D","year":"2000","journal-title":"Br J Haematol"},{"key":"CIT0087","doi-asserted-by":"crossref","first-page":"1084","DOI":"10.1046\/j.1365-2141.2001.02821-9.x","volume":"113","author":"vom Dahl S","year":"2001","journal-title":"Br J Haematol"},{"key":"CIT0088","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1097\/00125817-200211000-00005","volume":"4","author":"Grinzaid KA","year":"2002","journal-title":"Genet Med"},{"key":"CIT0089","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2007.02.057"},{"key":"CIT0090","doi-asserted-by":"crossref","first-page":"208","DOI":"10.1002\/ajh.21362","volume":"84","author":"Taddei TH","year":"2009","journal-title":"Am J Hematol"}],"container-title":["Expert Opinion on Pharmacotherapy"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.tandfonline.com\/doi\/pdf\/10.1517\/14656560903270520","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,5,22]],"date-time":"2020-05-22T03:39:44Z","timestamp":1590118784000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.tandfonline.com\/doi\/full\/10.1517\/14656560903270520"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2009,9,10]]},"references-count":87,"journal-issue":{"issue":"16","published-online":{"date-parts":[[2009,10,30]]},"published-print":{"date-parts":[[2009,11]]}},"alternative-id":["10.1517\/14656560903270520"],"URL":"https:\/\/doi.org\/10.1517\/14656560903270520","relation":{},"ISSN":["1465-6566","1744-7666"],"issn-type":[{"value":"1465-6566","type":"print"},{"value":"1744-7666","type":"electronic"}],"subject":[],"published":{"date-parts":[[2009,9,10]]}}}