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

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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Bibliographic Details
Main Authors: Anja F. Köhn, Lorenz Grigull, Marcel du Moulin, Sarah Kabisch, Luise Ammer, Cornelia Rudolph, Nicole M. Muschol
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Sanfilippo syndrome
Mucopolysaccharidosis type III
MPS IIIA
Stem cell transplantation
HSCT
Natural history
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426920300240

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http://www.sciencedirect.com/science/article/pii/S2214426920300240

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