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

Published 2020 View Full Article

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Title
Hematopoietic stem cell transplantation in mucopolysaccharidosis type IIIA: A case description and comparison with a genotype-matched control group
Authors
Keywords
Sanfilippo syndrome, Mucopolysaccharidosis type III, MPS IIIA, Stem cell transplantation, HSCT, Natural history
Journal
Molecular Genetics and Metabolism Reports
Volume 23, Issue -, Pages 100578
Publisher
Elsevier BV
Online
2020-03-24
DOI
10.1016/j.ymgmr.2020.100578

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