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Mucopolysaccharidosis II

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Internal Medicine

Idursulfase Extends Survival by a Decade in Mucopolysaccharidosis II: Final Results from the 18-Year Hunter Outcome Survey

The final report of the Hunter Outcome Survey (HOS) confirms that idursulfase treatment for Mucopolysaccharidosis II provides a 10-year survival benefit and a 57.9% reduction in mortality risk compared to untreated cohorts, alongside sustai

Dec 31, 2025
Neurology

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

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.

Dec 31, 2025Sign in to read

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