Impact of 144 weeks of laronidase therapy on body functions, endurance and general well-being in a Hurler-Scheie patient

J Inherit Metab Dis. 2006 Aug;29(4):590. doi: 10.1007/s10545-006-0329-5. Epub 2006 Jul 8.

Abstract

The quality of life of a Hurler-Scheie patient who experienced improvement in several organ functions and regained mobility after 144 weeks of laronidase enzyme replacement therapy is described.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Female
  • Humans
  • Iduronidase / administration & dosage
  • Iduronidase / therapeutic use*
  • Mucopolysaccharidosis I / drug therapy*
  • Mucopolysaccharidosis I / physiopathology
  • Quality of Life*
  • Recombinant Proteins / administration & dosage
  • Recombinant Proteins / therapeutic use

Substances

  • Recombinant Proteins
  • Iduronidase