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Long-term cognitive follow-up in children treated for Maroteaux-Lamy syndrome

BACKGROUND: It remains unclear to what extent the brain is affected by Maroteaux-Lamy syndrome (MPS VI), a progressive lysosomal storage disorder. While enzyme replacement therapy (ERT) elicits positive effects, the drug cannot cross the blood–brain barrier. We therefore studied cognitive developmen...

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Detalles Bibliográficos
Autores principales: Ebbink, Berendine J., Brands, Marion M. G., van den Hout, Johanna M. P., Lequin, Maarten H., van den Braak, Robert R. J. Coebergh, van de Weitgraven, Rianne L., Plug, Iris, Aarsen, Femke K., van der Ploeg, Ans T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Netherlands 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4754322/
https://www.ncbi.nlm.nih.gov/pubmed/26450354
http://dx.doi.org/10.1007/s10545-015-9895-8