Natural History and Galsulfase Treatment in Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) — 10 Year Follow-up of Patients Who Previously Participated in an MPS VI Survey Study

Mucopolysaccharidosis VI (MPS VI) is a clinically heterogeneous and progressive disorder with multiorgan manifestations caused by deficient N-acetlylgalactosamine-4-sulfatase activity. A cross-sectional Survey Study in individuals (n=121) affected with MPS VI was conducted between 2001–2002 to estab...

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Главные авторы: Giugliani, Roberto, Lampe, Christina, Guffon, Nathalie, Ketteridge, David, Teles, Elisa Leão, Wraith, James E., Jones, Simon A, Piscia-Nichols, Cheri, Lin, Ping, Quartel, Adrian, Harmatz, Paul
Формат: Artigo
Язык:Inglês
Опубликовано: 2014
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Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4107078/
https://ncbi.nlm.nih.gov/pubmed/24764221
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajmg.a.36584
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