{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T09:35:03Z","timestamp":1793180103374,"version":"4.5.0"},"reference-count":18,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2016,7,1]],"date-time":"2016-07-01T00:00:00Z","timestamp":1467331200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2016,7,1]],"date-time":"2016-07-01T00:00:00Z","timestamp":1467331200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2016,5,24]],"date-time":"2016-05-24T00:00:00Z","timestamp":1464048000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100007343","name":"Shire","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007343","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Genetics and Metabolism"],"published-print":{"date-parts":[[2016,7]]},"DOI":"10.1016\/j.ymgme.2016.05.006","type":"journal-article","created":{"date-parts":[[2016,5,10]],"date-time":"2016-05-10T20:31:33Z","timestamp":1462912293000},"page":"198-205","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":95,"title":["A phase 1\/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA"],"prefix":"10.1016","volume":"118","author":[{"given":"Simon A.","family":"Jones","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Breen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fiona","family":"Heap","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stewart","family":"Rust","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"de Ruijter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Evelien","family":"Tump","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan Pieter","family":"Marchal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luying","family":"Pan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yongchang","family":"Qiu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jou-Ku","family":"Chung","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nitin","family":"Nair","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick A.J.","family":"Haslett","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ann J.","family":"Barbier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frits A.","family":"Wijburg","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymgme.2016.05.006_bb0005","series-title":"The Metabolic and Molecular Bases of Inherited Disease","first-page":"3421","article-title":"The mucopolysaccharidoses","volume":"vol. III","author":"Neufeld","year":"2001"},{"key":"10.1016\/j.ymgme.2016.05.006_bb0010","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1186\/1750-1172-6-43","article-title":"Cognitive development in patients with mucopolysaccharidosis type III (Sanfilippo syndrome)","volume":"6","author":"Valstar","year":"2011","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.ymgme.2016.05.006_bb0015","doi-asserted-by":"crossref","first-page":"876","DOI":"10.1002\/ana.22092","article-title":"Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations","volume":"68","author":"Valstar","year":"2010","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymgme.2016.05.006_bb0020","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1007\/s13346-011-0043-1","article-title":"Safety evaluation of chronic intrathecal administration of heparan N-sulfatase in juvenile cynomolgus monkeys","volume":"2","author":"Pfeifer","year":"2012","journal-title":"Drug Deliv. Transl. Res."},{"key":"10.1016\/j.ymgme.2016.05.006_bb0025","doi-asserted-by":"crossref","first-page":"285","DOI":"10.4155\/bio.15.243","article-title":"A novel LC-MS\/MS assay for heparan sulfate screening in the cerebrospinal fluid of mucopolysaccharidosis IIIA patients","volume":"8","author":"Naimy","year":"2016","journal-title":"Bioanalysis"},{"key":"10.1016\/j.ymgme.2016.05.006_bb0030","series-title":"Vineland Adaptive Behavior Scales (Vineland\u2122-II)","author":"Sparrow","year":"2005"},{"key":"10.1016\/j.ymgme.2016.05.006_bb0035","series-title":"Bayley Scales of Infant and Toddler Development (Bayley-III)\u00ae","author":"Bayley","year":"2005"},{"key":"10.1016\/j.ymgme.2016.05.006_bb0040","series-title":"Kaufman Assessment Battery for Children","author":"Kaufman","year":"2004"},{"key":"10.1016\/j.ymgme.2016.05.006_bb0045","first-page":"129","article-title":"Methods of neurodevelopmental assessment in children with neurodegenerative disease: Sanfilippo syndrome","volume":"13","author":"Delaney","year":"2014","journal-title":"JIMD Rep."},{"key":"10.1016\/j.ymgme.2016.05.006_bb0050","series-title":"Glycosaminoglycans in Urine (Reference Range for Urine GAG Using a Colorimetric (1,9-Dimethyl-methylene Blue) Dye Binding Method)","year":"2014"},{"key":"10.1016\/j.ymgme.2016.05.006_bb0055","doi-asserted-by":"crossref","first-page":"209","DOI":"10.1016\/S0885-3924(99)00153-0","article-title":"A prospective study of catheter-related complications of intrathecal drug delivery systems","volume":"19","author":"Follett","year":"2000","journal-title":"J. Pain Symptom Manag."},{"key":"10.1016\/j.ymgme.2016.05.006_bb0060","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1097\/01.ajp.0000210955.93878.44","article-title":"Programmable intrathecal opioid delivery systems for chronic noncancer pain: a systematic review of effectiveness and complications","volume":"23","author":"Turner","year":"2007","journal-title":"Clin. J. Pain"},{"key":"10.1016\/j.ymgme.2016.05.006_bb0065","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1159\/000070870","article-title":"Complications of intrathecal baclofen pumps in children","volume":"39","author":"Gooch","year":"2003","journal-title":"Pediatr. Neurosurg."},{"key":"10.1016\/j.ymgme.2016.05.006_bb0070","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1542\/peds.2007-3847","article-title":"Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I","volume":"123","author":"Clarke","year":"2009","journal-title":"Pediatrics"},{"key":"10.1016\/j.ymgme.2016.05.006_bb0075","doi-asserted-by":"crossref","first-page":"533","DOI":"10.1016\/j.jpeds.2005.12.014","volume":"148","author":"Harmatz","year":"2006","journal-title":"J. Pediatr."},{"key":"10.1016\/j.ymgme.2016.05.006_bb0080","doi-asserted-by":"crossref","first-page":"465","DOI":"10.1097\/01.gim.0000232477.37660.fb","article-title":"A phase II\/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)","volume":"8","author":"Muenzer","year":"2006","journal-title":"Genet. Med."},{"key":"10.1016\/j.ymgme.2016.05.006_bb0085","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1016\/j.jpeds.2015.11.079","article-title":"A prospective natural history study of mucopolysaccharidosis type IIIA","volume":"170","author":"Shapiro","year":"2016","journal-title":"J. Pediatr."},{"key":"10.1016\/j.ymgme.2016.05.006_bb0090","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1186\/s13023-015-0265-2","article-title":"Immunogenicity of idursulfase and clinical outcomes in very young patients (16months to 7.5years) with mucopolysaccharidosis II (Hunter syndrome)","volume":"10","author":"Pano","year":"2015","journal-title":"Orphanet J. Rare Dis."}],"container-title":["Molecular Genetics and Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719216300646?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719216300646?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,21]],"date-time":"2025-10-21T23:22:11Z","timestamp":1761088931000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1096719216300646"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,7]]},"references-count":18,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2016,7]]}},"alternative-id":["S1096719216300646"],"URL":"https:\/\/doi.org\/10.1016\/j.ymgme.2016.05.006","relation":{},"ISSN":["1096-7192"],"issn-type":[{"value":"1096-7192","type":"print"}],"subject":[],"published":{"date-parts":[[2016,7]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"A phase 1\/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA","name":"articletitle","label":"Article Title"},{"value":"Molecular Genetics and Metabolism","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymgme.2016.05.006","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2016 The Authors. Published by Elsevier Inc.","name":"copyright","label":"Copyright"}]}}