Hunter syndrome is an exceedingly rare genetic disorder caused by a malfunctioning or missing enzyme. Patients with Hunter ...

A Denali Therapeutics drug for the rare enzyme deficiency Hunter syndrome is still in pivotal testing, but the company has guidance from the FDA on a pathway to get this therapy to the market sooner.

Denali Therapeutics Inc. announced positive long-term data from its Phase 1/2 study of tividenofusp alfa (DNL310) in patients with Hunter syndrome (MPS II), showing sustained reductions in key ...