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Musculoskeletal manifestations of mucopolysaccharidosis type VI and effects of enzyme replacement therapy

✍ Scribed by Marucha, Jolanta ;Jurecka, Agnieszka ;Różdżyńska-Świątkowska, Agnieszka ;Tylki-Szymańska, Anna

Book ID
111490131
Publisher
Walter de Gruyter GmbH
Year
2012
Tongue
English
Weight
1008 KB
Volume
7
Category
Article
ISSN
2391-5463

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## 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