Article
Niemann-Pick disease: sixteen-year follow-up of allogeneic bone marrow transplantation in a type B variant.
Journal of inherited metabolic disease - 1 Jan 2003
Victor S, Coulter J B S, Besley G T N, Ellis I, Desnick R J, Schuchman E H, Vellodi A
Abstract excerpt
Allogenic bone marrow transplantation (BMT) was carried out on a 3-year-old white caucasian girl with Niemann-Pick disease (NPD) type B. The donor was her unaffected brother. The patient was neurologically normal at the time of transplantation. Engraftment was based on cytogenetic studies and inc...
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