{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,20]],"date-time":"2026-02-20T16:04:00Z","timestamp":1771603440572,"version":"3.50.1"},"reference-count":14,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2017,11,1]],"date-time":"2017-11-01T00:00:00Z","timestamp":1509494400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":["clinicalkey.jp","clinicalkey.com","ejpn-journal.com","clinicalkey.es","clinicalkey.com.au","clinicalkey.fr","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["European Journal of Paediatric Neurology"],"published-print":{"date-parts":[[2017,11]]},"DOI":"10.1016\/j.ejpn.2017.07.015","type":"journal-article","created":{"date-parts":[[2017,7,29]],"date-time":"2017-07-29T14:45:56Z","timestamp":1501339556000},"page":"907-911","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":5,"title":["Limited benefits of presymptomatic cord blood transplantation in neurovisceral acid sphingomyelinase deficiency (ASMD) intermediate type"],"prefix":"10.1016","volume":"21","author":[{"given":"Oriane","family":"Mercati","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Samia","family":"Pichard","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marie","family":"Ouach\u00e9e","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Roseline","family":"Froissart","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Odile","family":"Fenneteau","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8877-2045","authenticated-orcid":false,"given":"Bastien","family":"Roche","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Monique","family":"Elmaleh-Berg\u00e8s","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yves","family":"Bertrand","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"H\u00e9l\u00e8ne","family":"Ogier de Baulny","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marie T.","family":"Vanier","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Manuel","family":"Schiff","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"issue":"3","key":"10.1016\/j.ejpn.2017.07.015_bib1","doi-asserted-by":"crossref","first-page":"206","DOI":"10.1016\/j.ymgme.2016.05.001","article-title":"Cause of death in patients with chronic visceral and chronic neurovisceral acid sphingomyelinase deficiency (Niemann-Pick disease type B and B variant): literature review and report of new cases","volume":"118","author":"Cassiman","year":"2016","journal-title":"Mol Genet Metab"},{"issue":"1","key":"10.1016\/j.ejpn.2017.07.015_bib2","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1186\/s13023-017-0572-x","article-title":"Disease manifestations and burden of illness in patients with acid sphingomyelinase deficiency (ASMD)","volume":"12","author":"McGovern","year":"2017","journal-title":"Orphanet J Rare Dis"},{"issue":"6","key":"10.1016\/j.ejpn.2017.07.015_bib3","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1007\/BF01800234","article-title":"Bone marrow transplantation for Niemann-Pick\u00a0type IA disease","volume":"15","author":"Bayever","year":"1992","journal-title":"J Inherit Metab Dis"},{"issue":"13","key":"10.1016\/j.ejpn.2017.07.015_bib4","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"},{"issue":"6610","key":"10.1016\/j.ejpn.2017.07.015_bib5","doi-asserted-by":"crossref","first-page":"1375","DOI":"10.1136\/bmj.295.6610.1375","article-title":"Treatment of Niemann-Pick disease type B by allogeneic bone marrow transplantation","volume":"295","author":"Vellodi","year":"1987","journal-title":"Br Med J Clin Res Ed"},{"issue":"8","key":"10.1016\/j.ejpn.2017.07.015_bib6","doi-asserted-by":"crossref","first-page":"775","DOI":"10.1023\/B:BOLI.0000009950.81514.c8","article-title":"Niemann-Pick disease: sixteen-year follow-up of allogeneic bone marrow transplantation in a type B variant","volume":"26","author":"Victor","year":"2003","journal-title":"J Inherit Metab Dis"},{"issue":"4","key":"10.1016\/j.ejpn.2017.07.015_bib7","doi-asserted-by":"crossref","first-page":"1022","DOI":"10.1542\/peds.2005-0867","article-title":"Successful hematopoietic stem cell transplantation for Niemann-Pick disease type B","volume":"116","author":"Shah","year":"2005","journal-title":"Pediatrics"},{"issue":"7","key":"10.1016\/j.ejpn.2017.07.015_bib8","doi-asserted-by":"crossref","first-page":"987","DOI":"10.1002\/pbc.21300","article-title":"Correction of enzyme levels with allogeneic hematopoeitic progenitor cell transplantation in Niemann-Pick type B","volume":"49","author":"Schneiderman","year":"2007","journal-title":"Pediatr Blood Cancer"},{"issue":"Pt 4","key":"10.1016\/j.ejpn.2017.07.015_bib9","first-page":"1050","article-title":"Highly variable neural involvement in sphingomyelinase-deficient Niemann-Pick disease caused by an ancestral Gypsy mutation","volume":"130","author":"Mihaylova","year":"2007","journal-title":"Brain J Neurol"},{"issue":"7","key":"10.1016\/j.ejpn.2017.07.015_bib10","doi-asserted-by":"crossref","first-page":"1117","DOI":"10.1002\/humu.21018","article-title":"Identification and characterization of SMPD1 mutations causing Niemann-Pick types A and B in Spanish patients","volume":"30","author":"Rodr\u00edguez-Pascau","year":"2009","journal-title":"Hum Mutat"},{"issue":"1\u20132","key":"10.1016\/j.ejpn.2017.07.015_bib11","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1016\/j.ymgme.2015.05.013","article-title":"Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency","volume":"116","author":"Wasserstein","year":"2015","journal-title":"Mol Genet Metab"},{"issue":"2","key":"10.1016\/j.ejpn.2017.07.015_bib12","doi-asserted-by":"crossref","first-page":"375","DOI":"10.1016\/j.jpeds.2012.07.052","article-title":"Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome","volume":"162","author":"Eisengart","year":"2013","journal-title":"J Pediatr"},{"issue":"2","key":"10.1016\/j.ejpn.2017.07.015_bib13","doi-asserted-by":"crossref","first-page":"349","DOI":"10.1016\/j.expneurol.2008.10.021","article-title":"Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease","volume":"215","author":"Dodge","year":"2009","journal-title":"Exp Neurol"},{"issue":"10","key":"10.1016\/j.ejpn.2017.07.015_bib14","doi-asserted-by":"crossref","first-page":"1893","DOI":"10.1038\/mt.2012.118","article-title":"Merits of combination cortical, subcortical, and cerebellar injections for the treatment of niemann-pick disease type A","volume":"20","author":"Bu","year":"2012","journal-title":"Mol Ther"}],"container-title":["European Journal of Paediatric Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1090379817316173?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1090379817316173?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2018,8,29]],"date-time":"2018-08-29T17:56:58Z","timestamp":1535565418000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1090379817316173"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2017,11]]},"references-count":14,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2017,11]]}},"alternative-id":["S1090379817316173"],"URL":"https:\/\/doi.org\/10.1016\/j.ejpn.2017.07.015","relation":{},"ISSN":["1090-3798"],"issn-type":[{"value":"1090-3798","type":"print"}],"subject":[],"published":{"date-parts":[[2017,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Limited benefits of presymptomatic cord blood transplantation in neurovisceral acid sphingomyelinase deficiency (ASMD) intermediate type","name":"articletitle","label":"Article Title"},{"value":"European Journal of Paediatric Neurology","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ejpn.2017.07.015","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2017 European Paediatric Neurology Society. Published by Elsevier Ltd. All rights reserved.","name":"copyright","label":"Copyright"}]}}