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Case Reports
. 1987 Nov 28;295(6610):1375-6.
doi: 10.1136/bmj.295.6610.1375.

Treatment of Niemann-Pick disease type B by allogeneic bone marrow transplantation

A Vellodi  1 , J R Hobbs, N M O'Donnell, B S Coulter, K Hugh-Jones
Affiliations
Case Reports

Treatment of Niemann-Pick disease type B by allogeneic bone marrow transplantation

A Vellodi et al. Br Med J (Clin Res Ed). .

Abstract

Allogenic bone marrow transplantation was carried out on a 3 year old girl with Niemann-Pick disease type B. Successful engraftment was achieved, and nine months after the procedure there was definite clearing of the sphingomyelin from the liver and pronounced clearing from the bone marrow. Any patient with Niemann-Pick disease type B complicated by early or severe hepatic impairment should be considered for bone marrow transplantation.

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