Article

Impact of ERT and Follow up of 17 Patients from the Same Family with Mild form of MPS II

2021-06-14

Abstract excerpt

<title>Abstract</title> <p><italic>Background:</italic> Mucopolysaccharidosis type II, also known as Hunter syndrome, is a rare X-linked recessive disorder caused by deficiency of the lysosomal enzyme iduronate-2- sulfatase (IDS), leading to progressive accumulation of glycosaminoglycans (GAGs) in several organs. Over the years, Enzyme Replacement Therapy (ERT) has provided significant benefit for patients, retar...

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Literature Corpus work: ef674ba3-ec80-5cc0-a638-a2da25599efe
DOI: 10.21203/rs.3.rs-540976/v1

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