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Hunter Syndrome Treatment: A Comprehensive Look at Evolving Care Approaches
Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), is a rare genetic disorder caused by a deficiency of the iduronate-2-sulfatase enzyme. This enzyme plays a crucial role in breaking down complex molecules called glycosaminoglycans (GAGs). When GAGs accumulate in the body, they lead to progressive damage affecting organs, tissues, and cognitive function. While the condition is lifelong, advancements in treatment have significantly improved the quality of life for many individuals.
One of the most widely used approaches in Hunter syndrome treatment is enzyme replacement therapy (ERT). This method involves infusing a synthetic version of the missing enzyme to help reduce GAG buildup. ERT has been shown to ease symptoms such as joint stiffness, breathing difficulties, and enlarged organs. Although it does not cross the blood-brain barrier—meaning it cannot directly address neurological symptoms—it remains a cornerstone therapy for physical…