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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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Detalles Bibliográficos
Autores principales:	Gaffke, Lidia, Pierzynowska, Karolina, Piotrowska, Ewa, Węgrzyn, Grzegorz
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Springer US 2017
Materias:	Review Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5769821/ https://www.ncbi.nlm.nih.gov/pubmed/28921412 http://dx.doi.org/10.1007/s11011-017-0111-4

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