Breakthrough in Hunter Syndrome: Brain-Penetrant Enzyme Therapy Stabilizes Cognitive Function and Clears Lysosomal Substrates
Highlights of the Study
- Tividenofusp alfa achieved a 91% reduction in cerebrospinal fluid (CSF) heparan sulfate levels, reaching levels comparable to unaffected children.
- Adaptive behavior, measured by the Vineland Adaptive Behavior Scales, remained stable or improved over a 157-week extension period.
- The engineered transferrin receptor-binding Fc domain successfully facilitated enzyme delivery across the blood-brain barrier.
- While infusion-related reactions were common, the safety profile supported long-term administration in pediatric patients.
Background: The Challenge of the Blood-Brain Barrier in MPS II
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This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.