{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,14]],"date-time":"2026-02-14T11:36:13Z","timestamp":1771068973728,"version":"3.50.1"},"reference-count":14,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[1995,7,1]],"date-time":"1995-07-01T00:00:00Z","timestamp":804556800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[1995,7]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>Neuropsychological assessment is essential in providing documentation of the untreated natural history of storage diseases associated with dementia and quantifying the effectiveness of treatment on central nervous system function. Baseline characterization and outcome of bone marrow transplantation (BMT) for three leukodystrophies and three mucopolysaccharidoses are presented. Results suggests that BMT for Hurler syndrome, adrenoleukodystrophy, and globoid cell leukodystrophy can be effective in preventing dementia if done early enough in the disease. Sanfilippo and Hunter syndromes do not benefit and BMT is not recommended. For metachromatic leukodystrophy, BMT is not recommended for symptomatic early\u2010onset forms of the disease. Further longitudinal follow\u2010up is needed to determine whether the benefits outweigh the risks of BMT for late\u2010onset and preclinical metachromatic leukodystrophy.<\/jats:p>","DOI":"10.1007\/bf00710053","type":"journal-article","created":{"date-parts":[[2004,11,30]],"date-time":"2004-11-30T18:52:45Z","timestamp":1101840765000},"page":"413-429","source":"Crossref","is-referenced-by-count":208,"title":["Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation"],"prefix":"10.1002","volume":"18","author":[{"given":"E. G.","family":"Shapiro","sequence":"first","affiliation":[{"name":"Department of Neurology University of Minnesota  Minneapolis Minnesota USA"},{"name":"Department of Pediatrics University of Minnesota  Minneapolis Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"L. A.","family":"Lockman","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Minnesota  Minneapolis Minnesota USA"},{"name":"Department of Pediatrics University of Minnesota  Minneapolis Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M.","family":"Balthazor","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Minnesota  Minneapolis Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"W.","family":"Krivit","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Minnesota  Minneapolis Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[1995,7]]},"reference":[{"key":"e_1_2_1_2_1","article-title":"Cognitive outcome of nine children with Sanfilippo syndrome after bone marrow transplantation and successful engraftment.","volume":"12","author":"Klein K","year":"1995","journal-title":"Bone Marrow Transplant"},{"key":"e_1_2_1_3_1","first-page":"203","volume-title":"Treatment of Genetic Diseases","author":"Krivit W","year":"1991"},{"key":"e_1_2_1_4_1","first-page":"883","volume-title":"Bone Marrow Transplanation","author":"Krivit W","year":"1994"},{"key":"e_1_2_1_5_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199001043220106"},{"key":"e_1_2_1_6_1","unstructured":"KrivitW ShapiroE LockmanL et al (1992)Recommendations for treatment of childhood cerebral form of adrenoleukodystrophy. InHobbsJR RichesPG eds.Correction of Certain Genetic Diseases by Transplantation 1991. The Cogent Trust 38\u201349.."},{"key":"e_1_2_1_7_1","article-title":"Bone marrow transplanation for Hurler syndrome.","volume":"12","author":"Krivit W","year":"1995","journal-title":"Bone Marrow Transplant"},{"key":"e_1_2_1_8_1","first-page":"1767","article-title":"Childhood cerebral form of adrenoleukodystrophy: short term effect of bone marrow transplantation on brain MR observations","volume":"15","author":"Loes DJ","year":"1994","journal-title":"Am J Neuroradiol"},{"key":"e_1_2_1_9_1","doi-asserted-by":"publisher","DOI":"10.1007\/BF01799621"},{"key":"e_1_2_1_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(06)80177-0"},{"key":"e_1_2_1_11_1","first-page":"119","volume-title":"Advances in Child Neuropsychology, Vol. III","author":"Shapiro E","year":"1993"},{"key":"e_1_2_1_12_1","first-page":"223","volume-title":"Treatment of Genetic Diseases","author":"Shapiro E","year":"1991"},{"key":"e_1_2_1_13_1","doi-asserted-by":"publisher","DOI":"10.1080\/01688639208402848"},{"key":"e_1_2_1_14_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.44.4.662"},{"key":"e_1_2_1_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.1320460222"}],"container-title":["Journal of Inherited Metabolic Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1007\/BF00710053","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1007\/BF00710053","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,15]],"date-time":"2023-09-15T00:11:39Z","timestamp":1694736699000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1007\/BF00710053"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[1995,7]]},"references-count":14,"journal-issue":{"issue":"4","published-print":{"date-parts":[[1995,7]]}},"alternative-id":["10.1007\/BF00710053"],"URL":"https:\/\/doi.org\/10.1007\/bf00710053","archive":["Portico"],"relation":{},"ISSN":["0141-8955","1573-2665"],"issn-type":[{"value":"0141-8955","type":"print"},{"value":"1573-2665","type":"electronic"}],"subject":[],"published":{"date-parts":[[1995,7]]}}}