An Analysis of Outpatient Healthcare Utilization and Costs in Children with Severe Mucopolysaccharidosis Type I in the United States
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DOI: 10.1016/j.jval.2018.04.902
Abstract: OBJECTIVE: Severe mucopolysaccharidosis (MPS) Type I (or Hurler syndrome) is a rare recessive genetic disorder caused by an enzyme deficiency. Without treatment (hematopoietic stem cell transplant and/or enzyme replacement therapy), children experience significant, rapid physical… read more here.
Keywords: severe mucopolysaccharidosis; utilization costs; outpatient healthcare; analysis ... See more keywords