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.
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