Outcomes of a Physician Survey on the Type, Progression, Assessment, and Treatment of Neurological Disease in Mucopolysaccharidoses
Abstract The mucopolysaccharidosis (MPS) disorders are a group of rare, inherited lysosomal storage disorders. In each of the 11 MPS (sub)types, deficiency in a specific lysosomal enzyme (1 of 11 identified enzymes) leads to accumulation of glycosaminoglycans, resulting in cell, tissue, and multi-or...
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Other Authors: | , , |
Format: | article |
Language: | eng |
Published: |
2018
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Subjects: | |
Online Access: | http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942018000100304 |
Country: | Brazil |
Oai: | oai:scielo:S2326-45942018000100304 |