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Renowned UNC Pediatric Biochemical Geneticist Leads First Study on a Novel Treatment for Hunter Syndrome

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic decline.The initial results are described in a new article published in the New England Journal of Medicine. The research assesses how an intravenous enzyme replacement therapy that crosses the blood-brain barrier can benefit individuals with Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.

January 6, 2026


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