Early HSCT corrects the skeleton in MPS.
In this issue of Blood, Pievani et al have identified a potential solution to the remaining barrier to the success of hematopoietic stem cell transplantation (HSCT) in children with severe phenotype Hurler syndrome (mucopolysaccharidosis type I [MPS I]).
DUKE UNIVERSITY MEDICAL CENTER.
Joanne
http://orcid.org/0000-0002-3370-0703
J
Kurtzberg
D000818:Animals; D001848:Bone Diseases, Developmental / Q000517:prevention & control*; D016026:Bone Marrow Transplantation / Q000379:methods*; D005260:Female; D006801:Humans; D008297:Male; D008059:Mucopolysaccharidosis I / Q000473:pathology* / Q000628:therapy*