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Growth charts for patients with Sanfilippo syndrome (Mucopolysaccharidosis type III)

BACKGROUND: Mucopolysaccharidosis (MPS) type III (Sanfilippo syndrome) comprises a group of rare, lysosomal storage diseases caused by the deficiency of one of four enzymes involved in the degradation of heparan sulfate. The clinical hallmark of the disease is severe neurological deterioration leadi...

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Detalles Bibliográficos
Autores principales:	Muschol, Nicole M., Pape, Daniel, Kossow, Kai, Ullrich, Kurt, Arash-Kaps, Laila, Hennermann, Julia B., Stücker, Ralf, Breyer, Sandra R.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2019
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6498678/ https://www.ncbi.nlm.nih.gov/pubmed/31046785 http://dx.doi.org/10.1186/s13023-019-1065-x

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