{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,29]],"date-time":"2025-10-29T13:35:20Z","timestamp":1761744920977,"version":"3.40.5"},"reference-count":10,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2019,6,1]],"date-time":"2019-06-01T00:00:00Z","timestamp":1559347200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":["clinicalkey.jp","clinicalkey.com","clinicalkey.es","clinicalkey.com.au","clinicalkey.fr","brainanddevelopment.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Brain and Development"],"published-print":{"date-parts":[[2019,6]]},"DOI":"10.1016\/j.braindev.2019.01.008","type":"journal-article","created":{"date-parts":[[2019,2,10]],"date-time":"2019-02-10T17:38:35Z","timestamp":1549820315000},"page":"546-550","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":8,"title":["Early enzyme replacement therapy enables a successful hematopoietic stem cell transplantation in mucopolysaccharidosis type IH: Divergent clinical outcomes in two Japanese siblings"],"prefix":"10.1016","volume":"41","author":[{"given":"Narutoshi","family":"Yamazaki","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Motomichi","family":"Kosuga","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kazuhiro","family":"Kida","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Go","family":"Takei","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4081-6739","authenticated-orcid":false,"given":"Yasuyuki","family":"Fukuhara","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hiroshi","family":"Matsumoto","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Masayoshi","family":"Senda","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Akihito","family":"Honda","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3896-5313","authenticated-orcid":false,"given":"Akira","family":"Ishiguro","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Takashi","family":"Koike","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hiromasa","family":"Yabe","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Torayuki","family":"Okuyama","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.braindev.2019.01.008_b0005","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1542\/peds.2008-0416","article-title":"Mucopolysaccharidosis I: management and treatment guidelines","volume":"123","author":"Muenzer","year":"2009","journal-title":"Pediatrics"},{"key":"10.1016\/j.braindev.2019.01.008_b0010","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.braindev.2019.01.008_b0015","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1016\/j.ymgme.2017.10.007","article-title":"Treatment of brain disease in the mucopolysaccharidoses","volume":"122","author":"Scarpa","year":"2017","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.braindev.2019.01.008_b0020","doi-asserted-by":"crossref","first-page":"1106","DOI":"10.1016\/j.bbmt.2015.02.011","article-title":"Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines","volume":"21","author":"Aldenhoven","year":"2015","journal-title":"Biol Blood Marrow Transplant"},{"key":"10.1016\/j.braindev.2019.01.008_b0025","doi-asserted-by":"crossref","first-page":"2164","DOI":"10.1182\/blood-2014-11-608075","article-title":"Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study","volume":"125","author":"Aldenhoven","year":"2015","journal-title":"Blood"},{"key":"10.1016\/j.braindev.2019.01.008_b0030","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1016\/j.braindev.2015.10.010","article-title":"Developmental assessment of VLBW infants at 18 months of age: A comparison study between KSPD and Bayley III","volume":"38","author":"Kono","year":"2016","journal-title":"Brain Dev"},{"key":"10.1016\/j.braindev.2019.01.008_b0035","first-page":"143","article-title":"Pre-transplant risk factors affecting outcome in Hurler syndrome","volume":"7","author":"Orchard","year":"2005","journal-title":"Genet Med"},{"key":"10.1016\/j.braindev.2019.01.008_b0040","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1097\/01.GIM.0000154299.22120.6A","article-title":"Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome","volume":"7","author":"Grewal","year":"2005","journal-title":"Genet Med"},{"key":"10.1016\/j.braindev.2019.01.008_b0045","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1186\/s13023-018-0816-4","article-title":"Status of newborn screening and follow up investigations for Mucopolysaccharidoses I and II in Taiwan","volume":"13","author":"Chuang","year":"2018","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.braindev.2019.01.008_b0050","doi-asserted-by":"crossref","first-page":"304","DOI":"10.1016\/j.ymgme.2016.05.015","article-title":"Pilot study of newborn screening for six lysosomal storage diseases using Tandem Mass Spectrometry","volume":"118","author":"Elliott","year":"2016","journal-title":"Mol Genet Metab"}],"container-title":["Brain and Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0387760418305412?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0387760418305412?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2019,4,24]],"date-time":"2019-04-24T19:00:19Z","timestamp":1556132419000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0387760418305412"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,6]]},"references-count":10,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2019,6]]}},"alternative-id":["S0387760418305412"],"URL":"https:\/\/doi.org\/10.1016\/j.braindev.2019.01.008","relation":{},"ISSN":["0387-7604"],"issn-type":[{"type":"print","value":"0387-7604"}],"subject":[],"published":{"date-parts":[[2019,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Early enzyme replacement therapy enables a successful hematopoietic stem cell transplantation in mucopolysaccharidosis type IH: Divergent clinical outcomes in two Japanese siblings","name":"articletitle","label":"Article Title"},{"value":"Brain and Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.braindev.2019.01.008","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2019 Published by Elsevier B.V. on behalf of The Japanese Society of Child Neurology.","name":"copyright","label":"Copyright"}]}}