{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,12]],"date-time":"2026-09-12T02:15:07Z","timestamp":1789179307165,"version":"build-2803163510"},"reference-count":95,"publisher":"Informa UK Limited","issue":"6","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Expert Review of Endocrinology &amp; Metabolism"],"published-print":{"date-parts":[[2011,11]]},"DOI":"10.1586\/eem.11.72","type":"journal-article","created":{"date-parts":[[2011,10,19]],"date-time":"2011-10-19T13:40:54Z","timestamp":1319031654000},"page":"755-768","source":"Crossref","is-referenced-by-count":3,"title":["Laronidase for the treatment of mucopolysaccharidosis type I"],"prefix":"10.1586","volume":"6","author":[{"given":"Lorne A","family":"Clarke","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"301","published-online":{"date-parts":[[2014,1,10]]},"reference":[{"key":"CIT0001","unstructured":"Neufeld EF, Muenzer J. The mucopolysaccharidoses. In:The Online Metabolic and Molecular Basis of Inherited Disease. Scriver CR, Beaudet AL, Sly WSet al.(Eds). McGraw-Hill, NY, USA, 1\u201373 (2004)."},{"key":"CIT0002","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1007\/s10545-008-1040-5","volume":"32","author":"Walkley","year":"2009","journal-title":"J. Inherit. Metab. Dis."},{"key":"CIT0003","unstructured":"Clarke LA. Mucopolysaccharidosis type I. In:GeneReviews. Pagon RA, Bird TD, Dolan CR, Stephens K (Eds). University of Washington, WA, USA (2007)."},{"key":"CIT0004","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1136\/adc.2007.135772","volume":"94","author":"Murphy","year":"2009","journal-title":"Arch. Dis. Child"},{"key":"CIT0005","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1007\/s004399900075","volume":"105","author":"Poorthuis","year":"1998","journal-title":"Hum. Genet."},{"key":"CIT0006","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1001\/jama.281.3.249","volume":"281","author":"Meikle","year":"1999","journal-title":"JAMA"},{"key":"CIT0007","doi-asserted-by":"crossref","first-page":"503","DOI":"10.1007\/s004390100606","volume":"109","author":"Beesley","year":"2001","journal-title":"Hum. Genet."},{"key":"CIT0008","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2004.12.006"},{"key":"CIT0009","first-page":"787","volume":"50","author":"Ashton","year":"1992","journal-title":"Am. J. Hum. Genet."},{"key":"CIT0010","doi-asserted-by":"crossref","first-page":"288","DOI":"10.1002\/humu.1380060403","volume":"6","author":"Scott","year":"1995","journal-title":"Hum. Mutat."},{"key":"CIT0011","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1002\/humu.20081","volume":"24","author":"Yogalingam","year":"2004","journal-title":"Hum. Mutat."},{"key":"CIT0012","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1146\/annurev.biochem.031209.093756","volume":"80","author":"Neufeld","year":"2010","journal-title":"Annu. Rev. Biochem."},{"key":"CIT0013","doi-asserted-by":"publisher","DOI":"10.1126\/science.162.3853.570"},{"key":"CIT0014","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1007\/BF01800340","volume":"15","author":"Muenzer","year":"1992","journal-title":"J. Inherit. Metab. Dis."},{"key":"CIT0015","doi-asserted-by":"crossref","first-page":"688","DOI":"10.1542\/peds.50.5.688","volume":"50","author":"Dekaban","year":"1972","journal-title":"Pediatrics"},{"key":"CIT0016","doi-asserted-by":"crossref","first-page":"1738","DOI":"10.1073\/pnas.68.8.1738","volume":"68","author":"Knudson","year":"1971","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"CIT0017","doi-asserted-by":"crossref","first-page":"303","DOI":"10.1073\/pnas.68.2.303","volume":"68","author":"Ferrante","year":"1971","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"CIT0018","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(81)91046-1"},{"key":"CIT0019","unstructured":"Edmunds T. B-glucocerebrosidase ceredase and cerezyme. In:Directory of Therapeutic Enzymes. McGrath BM, Walsh G (Eds). CRC Press, FL, USA, 117\u2013134 (2005)."},{"key":"CIT0020","doi-asserted-by":"crossref","first-page":"356","DOI":"10.1111\/j.1365-2141.2009.07974.x","volume":"148","author":"Prasad","year":"2010","journal-title":"Br. J. Haematol."},{"key":"CIT0021","doi-asserted-by":"crossref","first-page":"657","DOI":"10.1007\/s10545-011-9304-x","volume":"34","author":"van der Linden","year":"2011","journal-title":"J. Inherit. Metab. Dis."},{"key":"CIT0022","doi-asserted-by":"crossref","first-page":"485","DOI":"10.1016\/j.bbmt.2008.01.009","volume":"14","author":"Aldenhoven","year":"2008","journal-title":"Biol. Blood Marrow Transplant"},{"key":"CIT0023","doi-asserted-by":"crossref","first-page":"2601","DOI":"10.1182\/blood.V91.7.2601","volume":"91","author":"Peters","year":"1998","journal-title":"Blood"},{"key":"CIT0024","doi-asserted-by":"crossref","first-page":"1105","DOI":"10.1038\/sj.bmt.1704105","volume":"31","author":"Souillet","year":"2003","journal-title":"Bone Marrow Transplant"},{"key":"CIT0025","doi-asserted-by":"crossref","first-page":"1960","DOI":"10.1056\/NEJMoa032613","volume":"350","author":"Staba","year":"2004","journal-title":"N. Engl. J. Med."},{"key":"CIT0026","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1053\/j.seminhematol.2009.10.008","volume":"47","author":"Prasad","year":"2010","journal-title":"Semin. Hematol."},{"key":"CIT0027","doi-asserted-by":"crossref","first-page":"1239","DOI":"10.1038\/bmt.2009.319","volume":"45","author":"Orchard","year":"2010","journal-title":"Bone Marrow Transplant"},{"key":"CIT0028","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1186\/1750-1172-3-24","volume":"3","author":"Moore","year":"2008","journal-title":"Orphanet. J. Rare Dis."},{"key":"CIT0029","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1097\/00019052-199904000-00007","volume":"12","author":"Krivit","year":"2009","journal-title":"Curr. Opin. Neurol."},{"key":"CIT0030","doi-asserted-by":"crossref","first-page":"290","DOI":"10.1097\/00004703-200608000-00002","volume":"27","author":"Bjoraker","year":"2006","journal-title":"J. Dev. Behav. Pediatr."},{"key":"CIT0031","doi-asserted-by":"crossref","first-page":"618","DOI":"10.1016\/j.bbmt.2009.01.020","volume":"15","author":"Boelens","year":"2009","journal-title":"Biol. Blood Marrow Transplant"},{"key":"CIT0032","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1097\/01241398-200401000-00019","volume":"24","author":"Weisstein","year":"2004","journal-title":"J. Ped. Orthop."},{"key":"CIT0033","doi-asserted-by":"crossref","first-page":"1099","DOI":"10.1016\/S0161-6420(98)96014-6","volume":"105","author":"Gullingsrud","year":"2003","journal-title":"Ophthalmology"},{"key":"CIT0034","doi-asserted-by":"crossref","first-page":"882","DOI":"10.1016\/S0002-9149(03)00909-3","volume":"92","author":"Braunlin","year":"2003","journal-title":"Am. J. Cardiol."},{"key":"CIT0035","unstructured":"Kakkis E. \u03b1L-Iduronidase: The development of Aldurazyme (laronidase). In:Directory of Therapeutic Enzymes. McGrath BM, Walsh G (Eds). CRC Press, FL, USA, 239\u2013260 (2005)."},{"key":"CIT0036","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1006\/prep.1994.1035","volume":"5","author":"Kakkis","year":"1994","journal-title":"Protein Expr. Purif."},{"key":"CIT0037","doi-asserted-by":"crossref","first-page":"22758","DOI":"10.1074\/jbc.272.36.22758","volume":"272","author":"Zhao","year":"1997","journal-title":"J. Biol. Chem."},{"key":"CIT0038","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1042\/bj2590199","volume":"259","author":"Clements","year":"1989","journal-title":"Biochem. J."},{"key":"CIT0039","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1111\/j.1432-1033.1985.tb09158.x","volume":"152","author":"Clements","year":"1985","journal-title":"Eur. J. Biochem."},{"key":"CIT0040","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1111\/j.1432-1033.1985.tb09159.x","volume":"152","author":"Clements","year":"1985","journal-title":"Eur. J. Biochem."},{"key":"CIT0041","doi-asserted-by":"crossref","first-page":"899","DOI":"10.1042\/bj2820899","volume":"282","author":"Freeman","year":"1992","journal-title":"Biochem. J."},{"key":"CIT0042","doi-asserted-by":"publisher","DOI":"10.1017\/S1462399408000550"},{"key":"CIT0043","doi-asserted-by":"crossref","first-page":"112","DOI":"10.2353\/ajpath.2008.070564","volume":"172","author":"Simonaro","year":"2007","journal-title":"Am. J. Pathol."},{"key":"CIT0044","doi-asserted-by":"crossref","first-page":"35765","DOI":"10.1074\/jbc.M705124200","volume":"282","author":"Woloszynek","year":"2007","journal-title":"J. Biol. Chem."},{"key":"CIT0045","doi-asserted-by":"crossref","first-page":"317","DOI":"10.1165\/ajrcmb\/2.4.317","volume":"2","author":"Stahl","year":"1990","journal-title":"Am. J. Respir. Cell Mol. Biol."},{"key":"CIT0046","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1016\/0952-7915(92)90123-V","volume":"4","author":"Stahl","year":"1992","journal-title":"Curr. Opin. Immunol."},{"key":"CIT0047","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(78)90102-2"},{"key":"CIT0048","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1172\/JCI112262","volume":"77","author":"Kornfeld","year":"1985","journal-title":"J. Clin. Invest."},{"key":"CIT0049","doi-asserted-by":"crossref","first-page":"3083","DOI":"10.1073\/pnas.0308728100","volume":"101","author":"LeBowitz","year":"2004","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"CIT0050","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1016\/S0165-6147(03)00076-2","volume":"24","author":"Green","year":"2003","journal-title":"Trends Pharmacol. Sci."},{"key":"CIT0051","doi-asserted-by":"crossref","first-page":"202","DOI":"10.1038\/nrm1050","volume":"4","author":"Ghosh","year":"2003","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"CIT0052","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1042\/bj3040043","volume":"304","author":"Unger","year":"1994","journal-title":"Biochem. J."},{"key":"CIT0053","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.91.26.12937"},{"key":"CIT0054","doi-asserted-by":"publisher","DOI":"10.1006\/bmme.1996.0044"},{"key":"CIT0055","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2006-2156"},{"key":"CIT0056","doi-asserted-by":"crossref","first-page":"182","DOI":"10.1056\/NEJM200101183440304","volume":"344","author":"Kakkis","year":"2001","journal-title":"N. Eng. J. Med."},{"key":"CIT0057","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2006.08.007"},{"key":"CIT0058","doi-asserted-by":"crossref","first-page":"581","DOI":"10.1016\/j.jpeds.2004.01.046","volume":"144","author":"Wraith","year":"2004","journal-title":"J. Pediatr."},{"key":"CIT0059","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1542\/peds.2007-3847","volume":"123","author":"Clarke","year":"2009","journal-title":"Pediatrics"},{"key":"CIT0060","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1016\/j.ymgme.2008.10.009","volume":"96","author":"Giugliani","year":"2009","journal-title":"Mol. Genet. Metab."},{"key":"CIT0061","doi-asserted-by":"crossref","first-page":"2538","DOI":"10.1002\/ajmg.a.32294","volume":"146","author":"Munoz-Rojas","year":"2008","journal-title":"Am. J. Med. Genet."},{"key":"CIT0062","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2006.12.012"},{"key":"CIT0063","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0305480101"},{"key":"CIT0064","first-page":"2868","volume":"118","author":"Dickson","year":"2008","journal-title":"J. Clin. Invest."},{"key":"CIT0065","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1016\/j.jpeds.2008.07.004","volume":"154","author":"Wynn","year":"2009","journal-title":"J. Pediatr."},{"key":"CIT0066","doi-asserted-by":"crossref","first-page":"531","DOI":"10.1038\/sj.bmt.1705934","volume":"41","author":"Tolar","year":"2008","journal-title":"Bone Marrow Transplant"},{"key":"CIT0067","author":"V\u00e9lez-D\u00edaz-Pallar\u00e9s","year":"2011","journal-title":"Farm. Hosp."},{"key":"CIT0068","doi-asserted-by":"crossref","first-page":"215","DOI":"10.1016\/j.ymgme.2011.03.016","volume":"103","author":"Harada","year":"2011","journal-title":"Mol. Genet. Metab."},{"key":"CIT0069","first-page":"249","volume":"16","author":"Biernacka","year":"2010","journal-title":"Pediatr. Endocrinol. Diabetes Metab."},{"key":"CIT0070","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1016\/j.ymgme.2009.08.008","volume":"99","author":"Tylki-Szymanska","year":"2010","journal-title":"Mol. Genet. Metab."},{"key":"CIT0071","doi-asserted-by":"crossref","first-page":"e1242","DOI":"10.1542\/peds.2009-2843","volume":"126","author":"Valayannopoulos","year":"2010","journal-title":"Pediatrics"},{"key":"CIT0072","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1007\/s10545-010-9059-9","volume":"33","author":"Tylki-Szymanska","year":"2010","journal-title":"J. Inherit. Metab. Dis."},{"key":"CIT0073","doi-asserted-by":"crossref","first-page":"20","DOI":"10.1016\/j.ymgme.2009.12.019","volume":"100","author":"Valayannopoulos","year":"2010","journal-title":"Mol. Genet. Metab."},{"key":"CIT0074","doi-asserted-by":"crossref","first-page":"e183","DOI":"10.1542\/peds.2009-1728","volume":"125","author":"Gabrielli","year":"2010","journal-title":"Pediatrics"},{"key":"CIT0075","doi-asserted-by":"crossref","first-page":"230","DOI":"10.1007\/s10545-006-0237-8","volume":"29","author":"Anbu","year":"2010","journal-title":"J. Inherit. Metab. Dis."},{"key":"CIT0076","doi-asserted-by":"crossref","first-page":"590","DOI":"10.1007\/s10545-006-0329-5","volume":"29","author":"Soutar","year":"2006","journal-title":"J. Inherit. Metab. Dis."},{"key":"CIT0077","doi-asserted-by":"crossref","first-page":"821","DOI":"10.1007\/s10545-007-0551-9","volume":"30","author":"Arora","year":"2007","journal-title":"J. Inherit. Metab. Dis."},{"key":"CIT0078","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-007-0613-z"},{"key":"CIT0079","doi-asserted-by":"crossref","first-page":"1353","DOI":"10.1001\/archopht.125.10.1353","volume":"125","author":"Pitz","year":"2007","journal-title":"Arch. Ophthalmol."},{"key":"CIT0080","doi-asserted-by":"crossref","first-page":"1768","DOI":"10.1002\/ajmg.a.32317","volume":"146","author":"C\u00e1ceres-Marzal","year":"2008","journal-title":"Am. J. Med. Genet."},{"key":"CIT0081","first-page":"1057","author":"Yano","year":"2009","journal-title":"J. Inherit. Metab. Dis."},{"key":"CIT0082","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-006-0457-y"},{"key":"CIT0083","doi-asserted-by":"crossref","first-page":"727","DOI":"10.1007\/s00431-006-0316-8","volume":"166","author":"Tokic","year":"2007","journal-title":"Eur. J. Pediatr."},{"key":"CIT0084","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1002\/ajmg.a.31021","volume":"139","author":"Kloska","year":"2005","journal-title":"Am. J. Med. Genet."},{"key":"CIT0085","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2007.01.011"},{"key":"CIT0086","doi-asserted-by":"crossref","first-page":"1029","DOI":"10.1007\/s10545-011-9336-2","volume":"34","author":"Munoz-Rojas","year":"2011","journal-title":"J. Inherit. Metab. Dis."},{"key":"CIT0087","doi-asserted-by":"crossref","first-page":"984","DOI":"10.1007\/s10545-007-0686-8","volume":"30","author":"Cox-Brinkman","year":"2007","journal-title":"J. Inherit. Metab. Dis."},{"key":"CIT0088","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1177\/000456329403100309","volume":"31","author":"de Jong","year":"1994","journal-title":"Ann. Clin. Biochem."},{"key":"CIT0089","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1111\/j.1399-0004.2009.01324.x","volume":"77","author":"McGill","year":"2010","journal-title":"Clin. Genet."},{"key":"CIT0090","doi-asserted-by":"crossref","first-page":"457","DOI":"10.1097\/GIM.0b013e318211a7e1","volume":"13","author":"Wang","year":"2011","journal-title":"Genet. Med."},{"key":"CIT0091","doi-asserted-by":"crossref","first-page":"609","DOI":"10.1016\/j.jpeds.2008.11.005","volume":"154","author":"Wynn","year":"2009","journal-title":"J. Pediatr."},{"key":"CIT0092","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1016\/j.ymgme.2008.05.001","volume":"94","author":"Randall","year":"2008","journal-title":"Mol. Genet. Metab."},{"key":"CIT0093","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1007\/s10545-010-9254-8","volume":"34","author":"Langford-Smith","year":"2011","journal-title":"J. Inherit. Metab. Dis."},{"key":"CIT0094","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2006.01.005"},{"key":"CIT0095","author":"Clarke","year":"2011","journal-title":"J. Inherit. Metab. Dis."}],"container-title":["Expert Review of Endocrinology &amp; Metabolism"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.tandfonline.com\/doi\/pdf\/10.1586\/eem.11.72","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,12,11]],"date-time":"2021-12-11T21:06:36Z","timestamp":1639256796000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.tandfonline.com\/doi\/full\/10.1586\/eem.11.72"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2011,11]]},"references-count":95,"journal-issue":{"issue":"6","published-online":{"date-parts":[[2014,1,10]]},"published-print":{"date-parts":[[2011,11]]}},"alternative-id":["10.1586\/eem.11.72"],"URL":"https:\/\/doi.org\/10.1586\/eem.11.72","relation":{},"ISSN":["1744-6651","1744-8417"],"issn-type":[{"value":"1744-6651","type":"print"},{"value":"1744-8417","type":"electronic"}],"subject":[],"published":{"date-parts":[[2011,11]]}}}