Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines
Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines
C. Peters,C. Steward,Ibmtr and the Working Party on Inborn Errors of the Ebmt on behalf of the NMDP
2003 · DOI: 10.1038/sj.bmt.1703839
Bone Marrow Transplantation · 299 Citations
TLDR
The primary goals of HCT for selected inherited metabolic diseases have been to promote long-term survival with donor-derived engraftment and to optimize the quality of life.