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Sanfilippo syndrome: causes, consequences, and treatments
Sanfilippo syndrome, or mucopolysaccharidosis (MPS) type III, refers to one of five autosomal recessive, neurodegenerative lysosomal storage disorders (MPS IIIA to MPS IIIE) whose symptoms are caused by the deficiency of enzymes involved exclusively in heparan sulfate degradation. The primary charac...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Dove Medical Press
2015
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4664539/ https://www.ncbi.nlm.nih.gov/pubmed/26648750 http://dx.doi.org/10.2147/TACG.S57672 |