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Epidemiology of Mucopolysaccharidoses Update

10 Feb 2021

Abstract excerpt

Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders caused by a lysosomal enzyme deficiency or malfunction, which leads to the accumulation of glycosaminoglycans in tissues and organs. If not treated at an early stage, patients have various health problems, affecting their quality of life and life-span. Two therapeutic options for MPS are widely used in practice: enzyme replacement therapy and...

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PMID: 33578874
DOI: 10.3390/diagnostics11020273

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