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Breakthrough in Hunter Syndrome: Brain-Penetrant Enzyme Therapy Stabilizes Cognitive Function and Clears Lysosomal Substrates
Posted inNeurology news Pediatrics

Breakthrough in Hunter Syndrome: Brain-Penetrant Enzyme Therapy Stabilizes Cognitive Function and Clears Lysosomal Substrates

Posted by MedXY By MedXY 12/31/2025
A Phase 1-2 study of tividenofusp alfa reveals a 91% reduction in CSF heparan sulfate and stabilized adaptive behavior in children with MPS II, overcoming the blood-brain barrier via transferrin receptor-mediated transport.
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