Bone marrow transplantation for Niemann-Pick Type IA disease
β Scribed by E. Bayever; N. Kamani; P. Ferreira; G. A. Machin; M. Yudkoff; K. Conard; M. Palmieri; J. Radcliffe; D. A. Wenger; C. S. August
- Publisher
- Springer
- Year
- 1992
- Tongue
- English
- Weight
- 848 KB
- Volume
- 15
- Category
- Article
- ISSN
- 0141-8955
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π SIMILAR VOLUMES
## Abstract Liver homotransplantation was attempted as replacement therapy in a 2βyearβold patient with near total absence of sphingomyelinase activity typical of NiemannβPick disease type A. Satisfactory function of the graft was observed until the death of the recipient from respiratory complicat
Consideration of a bone marrow transplant (BMT) for a child with a genetic disease depends upon many factors including the pathophysiology of the disorder, the natural history of the disease, whether an alternative therapy exists and whether a donor is available. Children with disorders such as seve