Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase

BACKGROUND AND METHODS: Growth failure is characteristic of untreated mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome). Growth was studied in fifty-six MPS VI patients (5 to 29 years old) prior to and for up to 240 weeks of weekly infusions of recombinant human arylsulfatase B (rhASB)...

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Gorde:
Xehetasun bibliografikoak
Egile Nagusiak: Decker, Celeste, Yu, Zi-Fan, Giugliani, Roberto, Schwartz, Ida Vanessa D., Guffon, Nathalie, Teles, Elisa Leão, Miranda, M. Clara Sá, Wraith, J. Edmond, Beck, Michael, Arash, Laila, Scarpa, Maurizio, Ketteridge, David, Hopwood, John J., Plecko, Barbara, Steiner, Robert, Whitley, Chester B., Kaplan, Paige, Swiedler, Stuart J., Conrad, Susan, Harmatz, Paul
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: 2010
Gaiak:
Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC2904323/
https://ncbi.nlm.nih.gov/pubmed/20634905
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3233/PRM-2010-0113
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