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Novel therapies for mucopolysaccharidosis type III

Mucopolysaccharidosis type III (MPS III) or Sanfilippo disease is an orphan inherited lysosomal storage disease and one of the most common MPS subtypes. The classical presentation is an infantile‐onset neurodegenerative disease characterised by intellectual regression, behavioural and sleep disturba...

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Detalles Bibliográficos
Autores principales:	Seker Yilmaz, Berna, Davison, James, Jones, Simon A., Baruteau, Julien
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons, Inc. 2020
Materias:	Review Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8436764/ https://www.ncbi.nlm.nih.gov/pubmed/32944950 http://dx.doi.org/10.1002/jimd.12316

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