Article

The Landscape of Mucopolysaccharidosis in Southern and Eastern European Countries: A Survey from 19 Specialistic Centers.

2021-11-19

Abstract excerpt

<title>Abstract</title> <p><bold>Background: </bold>Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders caused by defects in genes coding for different lysosomal enzymes which degrade glycosaminoglycans. Impaired lysosomal degradation causes cell dysfunction leading to progressive multiorgan involvement, disabling consequences and poor life expectancy. Enzyme replacement therapy (ERT) is now av...

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Literature Corpus work: 4c62fa20-143f-564a-ba1b-39781f7ada59
DOI: 10.21203/rs.3.rs-1071210/v1

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