Tag: enzyme replacement therapy (ERT)

Abusive head trauma results in fewer productive life-years

Brain-penetrant enzyme replacement therapy improves biomarkers in mucopolysaccharidosis type II

byZhenyu Liand1 others

January 14, 2026

1. In this open-label phase 1–2 study, pediatric patients with mucopolysaccharidosis type II treated with tividenofusp alfa showed substantial reductions ...

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