Journal
PEDIATRICS
Volume 116, Issue 4, Pages 1022-1025Publisher
AMER ACAD PEDIATRICS
DOI: 10.1542/peds.2005-0867
Keywords
hematopoietic stem cell transplantation; Niemann-Pick disease
Categories
Funding
- NCRR NIH HHS [M01 RR00043] Funding Source: Medline
Ask authors/readers for more resources
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.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available