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Long-term outcomes in MPS-IH: throwing stars

Michael A Pulsipher1

  • 1HUNTSMAN CANCER INSTITUTE; UNIVERSITY OF UTAH SCHOOL OF MEDICINE; PRIMARY CHILDREN'S HOSPITAL.

Blood
|March 28, 2015
PubMed

After a herculean data-gathering effort, in this issue of Blood, Aldenhoven and colleagues from Europe and North America provide an eye-opening assessment of long-term neurocognitive, organ, joint, and tissue function after allogeneic transplantation of children with mucopolysaccharidosis type I–Hurler syndrome (MPS-IH), along with an analysis defining a path to better these outcomes.

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