Hematopoietic cell transplantation in inborn errors of metabolism

Although certain inborn errors improve after stem cell transplantation, the mechanisms by which these beneficial effects are mediated, particularly in central nervous system disease, need further investigation. Long-term follow-up in children with metabolic disorders is essential to define the role of stem cell transplantation in the different diseases, and to identify novel and thus far unknown long-term consequences of the respective errors of metabolism.