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Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme(®)) therapy

Characteristic cardiac valve abnormalities and left ventricular hypertrophy are present in untreated patients with mucopolysaccharidosis type VI (MPS VI). Cardiac ultrasound was performed to investigate these findings in subjects during long-term enzyme replacement therapy (ERT) with recombinant hum...

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Autores principales: Braunlin, E., Rosenfeld, H., Kampmann, C., Johnson, J., Beck, M., Giugliani, R., Guffon, N., Ketteridge, D., Sá Miranda, C. M., Scarpa, M., Schwartz, I. V., Leão Teles, E., Wraith, J. E., Barrios, P., Dias da Silva, E., Kurio, G., Richardson, M., Gildengorin, G., Hopwood, J. J., Imperiale, M., Schatz, A., Decker, C., Harmatz, P.
Formato: Artigo
Lenguaje:Inglês
Publicado: Springer Netherlands 2012
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3590402/
https://ncbi.nlm.nih.gov/pubmed/22669363
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-012-9481-2
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