Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism
Published 2019 View Full Article
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Title
Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism
Authors
Keywords
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Journal
Frontiers in Pediatrics
Volume 7, Issue -, Pages -
Publisher
Frontiers Media SA
Online
2019-10-25
DOI
10.3389/fped.2019.00433
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- Mucopolysaccharidosis I: Management and Treatment Guidelines
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