Clinical Data49-week data from the ongoing open-label Phase I/II trial of DNL126 are consistent with previously announced data, including normalization of CSF HS levels.
Market CompetitionSwitching MPS II patients from the existing drug should be much faster than normal, especially in children, as the clinical program was based on switches.
Regulatory PathwayDenali must navigate the challenges which Elaprase faced with a more sophisticated launch strategy for tividenofusp alfa.