{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,13]],"date-time":"2026-02-13T17:03:24Z","timestamp":1771002204481,"version":"3.50.1"},"reference-count":10,"publisher":"American Academy of Pediatrics (AAP)","issue":"4","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2005,10,1]]},"abstract":"<jats:p>Histocompatible hematopoietic stem cell transplantation (HSCT) was conducted on a 4.5-year-old girl with Niemann-Pick disease type B. The donor was her unaffected brother. At the time of transplantation, she had severe pulmonary disease. After her first HSCT, she developed graft failure. Five years after her second HSCT, her sphingomyelinase levels are within normal levels, she has no pulmonary symptoms, and aside from persistent graft versus host disease, she is doing well.<\/jats:p>","DOI":"10.1542\/peds.2005-0867","type":"journal-article","created":{"date-parts":[[2005,9,30]],"date-time":"2005-09-30T18:42:55Z","timestamp":1128105775000},"page":"1022-1025","source":"Crossref","is-referenced-by-count":31,"title":["Successful Hematopoietic Stem Cell Transplantation for Niemann-Pick Disease Type B"],"prefix":"10.1542","volume":"116","author":[{"given":"Ami J.","family":"Shah","sequence":"first","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation"},{"name":"Department of Pediatrics, Keck School of Medicine"},{"name":"Saban Research Institute"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Neena","family":"Kapoor","sequence":"additional","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation"},{"name":"Department of Pediatrics, Keck School of Medicine"},{"name":"Saban Research Institute"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gay M.","family":"Crooks","sequence":"additional","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation"},{"name":"Department of Pediatrics, Keck School of Medicine"},{"name":"Saban Research Institute"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Robertson","family":"Parkman","sequence":"additional","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation"},{"name":"Department of Pediatrics, Keck School of Medicine"},{"name":"Saban Research Institute"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kenneth I.","family":"Weinberg","sequence":"additional","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation"},{"name":"Department of Pediatrics, Keck School of Medicine"},{"name":"Saban Research Institute"},{"name":"General Clinical Research Center, Childrens Hospital Los Angeles, Los Angeles, California"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kathy","family":"Wilson","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Keck School of Medicine"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Donald B.","family":"Kohn","sequence":"additional","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation"},{"name":"Department of Pediatrics, Keck School of Medicine"},{"name":"Saban Research Institute"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"417","published-online":{"date-parts":[[2005,10,1]]},"reference":[{"key":"2021120819493491600_R1","unstructured":"Schuchman EH, Desnick RJ. Niemann Pick disease type A and B: acid sphyngomyelinase deficiencies. In: Scriver CR, Beaudet AL, Sly WS, Valle D, eds. The Metabolic and Molecular Basis of Inherited Disease. 8th ed. New York, NY: McGraw-Hill; 2001"},{"key":"2021120819493491600_R2","doi-asserted-by":"crossref","unstructured":"Elleder M, Cihula J. Niemann-Pick disease (variation in the sphingomyelinase deficient group). Neurovisceral phenotype (A) with an abnormally protracted clinical course and variable expression of neurological symptomatology in three siblings. Eur J Pediatr.\u20081983;140:323\u2013328","DOI":"10.1007\/BF00442673"},{"key":"2021120819493491600_R3","doi-asserted-by":"crossref","unstructured":"Elleder M, Nevoral J, Spicakova V, et al. A new variant of sphingomyelinase deficiency (Niemann-Pick): visceromegaly, minimal neurological lesions and low in vivo degradation rate of sphingomyelin. 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Gene Ther.\u20082000;7:1768\u20131776","DOI":"10.1038\/sj.gt.3301300"}],"container-title":["Pediatrics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/publications.aap.org\/pediatrics\/article-pdf\/116\/4\/1022\/1024193\/zpe01005001022.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/publications.aap.org\/pediatrics\/article-pdf\/116\/4\/1022\/1024193\/zpe01005001022.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,12,8]],"date-time":"2021-12-08T22:51:15Z","timestamp":1639003875000},"score":1,"resource":{"primary":{"URL":"https:\/\/publications.aap.org\/pediatrics\/article\/116\/4\/1022\/68079\/Successful-Hematopoietic-Stem-Cell-Transplantation"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2005,10,1]]},"references-count":10,"journal-issue":{"issue":"4","published-online":{"date-parts":[[2005,10,1]]},"published-print":{"date-parts":[[2005,10,1]]}},"URL":"https:\/\/doi.org\/10.1542\/peds.2005-0867","relation":{},"ISSN":["0031-4005","1098-4275"],"issn-type":[{"value":"0031-4005","type":"print"},{"value":"1098-4275","type":"electronic"}],"subject":[],"published":{"date-parts":[[2005,10,1]]}}}