Idursulfase and Beyond: Therapeutic Advances in Hunter Syndrome

bir ses eklendi Health

2025-06-17 10:35:06

Hunter Syndrome, also known as Mucopolysaccharidosis Type II (MPS II), is a rare X-linked lysosomal storage disorder caused by mutations in the IDS gene, leading to a deficiency of the enzyme iduronate-2-sulfatase. This enzyme is critical for breaking down glycosaminoglycans (GAGs) like dermatan sulfate and heparan sulfate. Without it, GAGs accumulate in tissues, triggering progressive damage...

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