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How close are we to therapies for Sanfilippo disease?
Sanfilippo disease is one of mucopolysaccharidoses (MPS), a group of lysosomal storage diseases characterized by accumulation of partially degraded glycosaminoglycans (GAGs). It is classified as MPS type III, though it is caused by four different genetic defects, determining subtypes A, B, C and D....
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| Publicado no: | Metab Brain Dis |
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| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Springer US
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5769821/ https://ncbi.nlm.nih.gov/pubmed/28921412 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s11011-017-0111-4 |
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