Current and new therapies for mucopolysaccharidoses
The mucopolysaccharidoses (MPSs) are a subset of lysosomal storage diseases caused by deficiencies in the enzymes required to metabolize glycosaminoglycans (GAGs), a group of extracellular heteropolysaccharides that play diverse roles in human physiology. As a result, GAGs accumulate in multiple tis...
Main Authors: | Monica Penon-Portmann, David R. Blair, Paul Harmatz |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2023-02-01
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Series: | Pediatrics and Neonatology |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S187595722200225X |
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