Effects of enzyme replacement therapy on growth in patients with mucopolysaccharidosis type II
β Scribed by G. Schulze-Frenking; Simon A. Jones; J. Roberts; M. Beck; J. E. Wraith
- Publisher
- Springer
- Year
- 2010
- Tongue
- English
- Weight
- 134 KB
- Volume
- 34
- Category
- Article
- ISSN
- 0141-8955
No coin nor oath required. For personal study only.
π SIMILAR VOLUMES
## Communicated by Andreas Gal Mucopolysaccharidosis type VI (MPS VI), or Maroteaux-Lamy syndrome, is a lysosomal storage disorder caused by a deficiency of N-acetylgalactosamine-4-sulfatase (ARSB). Seven MPS VI patients were chosen for the initial clinical trial of enzyme replacement therapy. Dir
Mucopolysaccharidosis type I (MPS I) is an autosomal recessive lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase (IDUA). Mutations in the gene are responsible for the enzyme deficiency, which leads to the intralysosomal storage of the partially degraded glycosaminoglycans derm