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Hematopoietic stem cell transplantation in mucopolysaccharidosis type IIIA: A case description and comparison with a genotype-matched control group

BACKGROUND: Mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo A syndrome) is a chronic progressive neurodegenerative storage disorder caused by a deficiency of lysosomal sulfamidase. The clinical hallmarks are sleep disturbances, behavioral abnormalities and loss of cognitive, speech and motor a...

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Detalles Bibliográficos
Autores principales:	Köhn, Anja F., Grigull, Lorenz, du Moulin, Marcel, Kabisch, Sarah, Ammer, Luise, Rudolph, Cornelia, Muschol, Nicole M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2020
Materias:	Research Paper
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7093801/ https://www.ncbi.nlm.nih.gov/pubmed/32226768 http://dx.doi.org/10.1016/j.ymgmr.2020.100578

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