[Overview of enzyme replacement therapy in mucopolysaccharidosis]

Presse Med. 2007 Mar:36 Spec No 1:1S96-9.
[Article in French]

Abstract

Mucopolysaccharidosis are rare, multisystemic and progressive diseases with an extremely various clinical spectrum. For the type I, II and VI mucopolysaccharidosis, enzyme replacement therapy is available. In these three diseases, enzyme replacement therapy induces a reduction in urinary glycosaminoglycanes excretion and on improvement in functional tests, 6 minutes walk test, pulmonary function test and range of motion.

Publication types

  • English Abstract
  • Review

MeSH terms

  • Glycosaminoglycans / urine
  • Humans
  • Iduronate Sulfatase / therapeutic use*
  • Iduronidase / therapeutic use*
  • Mucopolysaccharidoses / therapy*
  • N-Acetylgalactosamine-4-Sulfatase / therapeutic use*
  • Recombinant Proteins / therapeutic use

Substances

  • Glycosaminoglycans
  • Recombinant Proteins
  • N-Acetylgalactosamine-4-Sulfatase
  • galsulfase
  • Iduronate Sulfatase
  • Iduronidase