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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...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer Netherlands
2015
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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 |