Early HSCT corrects the skeleton in MPS.

Blood
Joanne Kurtzberg

Abstract

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]).

References

May 7, 2004·The New England Journal of Medicine·Susan L StabaJoanne Kurtzberg
Apr 14, 2009·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Jaap Jan BoelensUNKNOWN EUROCORD, Inborn error Working Party of EBMT and Duke University
Mar 16, 2013·Blood·Jaap Jan BoelensUNKNOWN Centre for International Blood and Marrow Research

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Citations

Dec 24, 2015·Frontiers in Public Health·Dora Il'yasovaAlexander Kinev
Nov 22, 2017·Expert Opinion on Orphan Drugs·Molly StapletonShunji Tomatsu

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