{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,3]],"date-time":"2026-08-03T17:17:52Z","timestamp":1785777472056,"version":"3.56.0"},"reference-count":14,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2014,2,1]],"date-time":"2014-02-01T00:00:00Z","timestamp":1391212800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Transplantation"],"published-print":{"date-parts":[[2014,5]]},"abstract":"Abstract<\/jats:title>Hurler syndrome type 1 (MPS<\/jats:styled-content>\u20101) is an autosomal recessive lysosomal disorder due to the deficiency of the enzyme alpha\u2010L\u2010iduronidase which is necessary for the degradation of dermatan and heparan sulfate. It is characterized by deposit of glycosaminoglycans in tissues, progressive multisystem dysfunction, and early death. HSCT<\/jats:styled-content> for children with MPS<\/jats:styled-content>\u2010I is effective, resulting in increased life expectancy and improvement of clinical parameters. The spinal MRI<\/jats:styled-content> performed on a female 10\u00a0yr old undergoing HSCT<\/jats:styled-content> at the age of 18\u00a0months and receiving ERT<\/jats:styled-content> revealed a considerable decrease in soft tissue around the tip of odontoid causing a significant reduction in spinal cord compression. In light of this result, we suppose that combined ERT<\/jats:styled-content> and HSCT<\/jats:styled-content> are successful in Hurler I disease.<\/jats:p>","DOI":"10.1111\/petr.12231","type":"journal-article","created":{"date-parts":[[2014,2,1]],"date-time":"2014-02-01T11:05:14Z","timestamp":1391252714000},"source":"Crossref","is-referenced-by-count":9,"title":["Hematopoietic stem cell transplantation effects on spinal cord compression in Hurler"],"prefix":"10.1111","volume":"18","author":[{"given":"Giovanna","family":"Ferrara","sequence":"first","affiliation":[{"name":"University of Trieste Trieste Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalia","family":"Maximova","sequence":"additional","affiliation":[{"name":"Institute for Maternal and Child Health IRCCS Burlo Garofolo Trieste Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Floriana","family":"Zennaro","sequence":"additional","affiliation":[{"name":"Institute for Maternal and Child Health IRCCS Burlo Garofolo Trieste Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Massimo","family":"Gregori","sequence":"additional","affiliation":[{"name":"Institute for Maternal and Child Health IRCCS Burlo Garofolo Trieste Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paolo","family":"Tamaro","sequence":"additional","affiliation":[{"name":"Institute for Maternal and Child Health IRCCS Burlo Garofolo University of Trieste Trieste Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2014,2]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.69.8.2048"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM197101282840425"},{"key":"e_1_2_9_4_1","first-page":"3421","volume-title":"The Metabolic and Molecular Basis of Inherited Disease","author":"Neufeld EF","year":"2001"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pcl.2009.11.004"},{"key":"e_1_2_9_6_1","doi-asserted-by":"crossref","first-page":"2601","DOI":"10.1182\/blood.V91.7.2601","article-title":"Hurler syndrome. II. Outcome of HLA genotypically identical sibling and HLA\u2010haploidentical related donor bone marrow transplantation in fifty\u2010four children. The Storage Disease Collaborative Study Group","volume":"91","author":"Peters C","year":"1998","journal-title":"Blood"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V87.11.4894.bloodjournal87114894"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2008.01.009"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1136\/ard.2008.095315"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10067-006-0299-x"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-005-0136-4"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200101183440304"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1517\/14656566.6.3.489"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2012.09.004"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bone.2013.03.014"}],"container-title":["Pediatric Transplantation"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1111%2Fpetr.12231","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/petr.12231","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,29]],"date-time":"2023-09-29T15:06:38Z","timestamp":1695999998000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/petr.12231"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2014,2]]},"references-count":14,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2014,5]]}},"alternative-id":["10.1111\/petr.12231"],"URL":"https:\/\/doi.org\/10.1111\/petr.12231","archive":["Portico"],"relation":{},"ISSN":["1397-3142","1399-3046"],"issn-type":[{"value":"1397-3142","type":"print"},{"value":"1399-3046","type":"electronic"}],"subject":[],"published":{"date-parts":[[2014,2]]}}}