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Early HSCT corrects the skeleton in MPS.

Joanne Kurtzberg
Published in: Blood (2015)
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]).
Keyphrases
  • hematopoietic stem cell
  • young adults
  • acute myeloid leukemia
  • replacement therapy
  • early onset
  • human health
  • climate change
  • solid state