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Replacement therapy in Mucopolysaccharidosis type VI: advantages of early onset of therapy

โœ Scribed by Dyane Auclair; John J. Hopwood; Douglas A. Brooks; Jeffrey F. Lemontt; Allison C. Crawley


Book ID
117735486
Publisher
Elsevier Science
Year
2003
Tongue
English
Weight
283 KB
Volume
78
Category
Article
ISSN
1096-7192

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โœ L. Karageorgos; P. Harmatz; J. Simon; A. Pollard; P. R. Clements; D. A. Brooks; ๐Ÿ“‚ Article ๐Ÿ“… 2004 ๐Ÿ› John Wiley and Sons ๐ŸŒ English โš– 121 KB ๐Ÿ‘ 1 views

## 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