{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,7]],"date-time":"2026-04-07T08:28:02Z","timestamp":1775550482828,"version":"3.50.1"},"reference-count":23,"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>Within the past decade, bone marrow transplantation has been applied to over 200 patients worldwide with the intention of treating storage diseases. Bone marrow transplantation has provided a method for treatment of adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome. After engraftment, significant improvement in the clinical course of each of these diseases occurs. Survival data of engrafted patients are superior to those of non\u2010transplanted. Engraftment and the resulting enzymatic reconstitution are concordant. Outcomes based on neuropsychological tests indicate continued maintenance and in some cases increase in cognitive function. Magnetic resonance imaging as well as spectroscopic examinations of the brain provide further evidence that positive changes occur in the central nervous system following long\u2010term engraftment. A better quality of life follows engraftment. Greater gains from use of bone marrow transplantation for these particular storage diseases will occur in the future. Earlier diagnosis will allow bone marrow transplantation in the presymptomatic stage at a younger age, providing an enhancement of positive effects noted from such treatment. At the same time, advances in bone marrow technology will serve to reduce the risk factors involved with the bone marrow transplantation process itself. These two factors taken together will be more than additive in providing benefits from use of bone marrow transplantation.<\/jats:p>","DOI":"10.1007\/bf00710052","type":"journal-article","created":{"date-parts":[[2004,11,30]],"date-time":"2004-11-30T18:52:45Z","timestamp":1101840765000},"page":"398-412","source":"Crossref","is-referenced-by-count":126,"title":["The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome"],"prefix":"10.1002","volume":"18","author":[{"given":"W.","family":"Krivit","sequence":"first","affiliation":[{"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 and Pediatrics University of Minnesota  Minneapolis Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"P. A.","family":"Watkins","sequence":"additional","affiliation":[{"name":"Division of Neurogenetics, Kennedy\u2010Krieger Institute Johns Hopkins University  Baltimore Maryland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J.","family":"Hirsch","sequence":"additional","affiliation":[{"name":"Division of Audiology, Audiology Clinic, Department of Otolaryngology University of Minnesota  Minneapolis Minnesota USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"E. G.","family":"Shapiro","sequence":"additional","affiliation":[{"name":"Department of Neurology and 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","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199006283222607"},{"key":"e_1_2_1_3_1","doi-asserted-by":"publisher","DOI":"10.1001\/archneur.1992.00530340114026"},{"key":"e_1_2_1_4_1","unstructured":"KatoK InabaJ HasegawaS(1994)Four cases of bone marrow transplantation for metabolic diseases. Bone marrow transplantation in children: current results and controversies.Hilton Head Conference Iowa School of Medicine Abstract 28.."},{"key":"e_1_2_1_5_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI110963"},{"key":"e_1_2_1_6_1","volume-title":"Principles of Clinical Immunology, vol. 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Transplantation in children. 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