Rapid deterioration of a patient with mucopolysaccharidosis type I during interruption of enzyme replacement therapy

Am J Med Genet A. 2007 Aug 15;143A(16):1925-7. doi: 10.1002/ajmg.a.31831.
No abstract available

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Child, Preschool
  • Female
  • Hair / ultrastructure
  • Humans
  • Iduronidase / therapeutic use*
  • Microscopy, Electron, Scanning
  • Mucopolysaccharidosis I / drug therapy*
  • Mucopolysaccharidosis I / pathology
  • Treatment Outcome

Substances

  • Iduronidase