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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Bibliographic Details
Main Author: Scarpa,Maurizio (author)
Other Authors: Harmatz,Paul R. (author), Meesen,Bianca (author), Giugliani,Roberto (author)
Format: article
Language:eng
Published: 2018
Subjects:
Online Access:http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942018000100304
Country:Brazil
Oai:oai:scielo:S2326-45942018000100304