Tividenofusp alfa treatment showed reduction and normalization in key disease biomarkers, stabilization or improvement in clinical endpoints including adaptive behavior, cognition and hearing, and normalization of liver volume Most common treatment-related adverse events were infusion-related reactions, which decreased with continued useTividenofusp alfa is an investigational, next-generation enzyme replacement therapy engineered to cross the blood-brain barrierBiologics License Application for tividenofusp ...