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Article

Mucopolysaccharidosis VII in Brazil: Natural History and Clinical Findings

2021-03-18

Abstract excerpt

<title>Abstract</title> <p><bold>Background: </bold>Mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome, caused by deficiency of the lysosomal enzyme β-glucuronidase, is an ultra-rare disorder with scarce epidemiological data and few publications about natural history and clinical spectrum. <bold>Methods:</bold> We conducted a case series report which included retrospective data from all MPS VII...

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Literature Corpus work
8fb529ad-1c80-53f7-ad06-980bd8fb8ff2
DOI
10.21203/rs.3.rs-315078/v1
Open publication

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Mucopolysaccharidosis VII in Brazil: Natural History and Clinical FindingsDOI 10.21203/rs.3.rs-315078/v1
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