Denali Therapeutics Reports Positive Three-Month Data from Phase 1/2 Study with ETV:IDS (DNL310) in Patients with Hunter Syndrome (MPS II)
Sustained normalization and further decreased levels of CSF glycosaminoglycan heparan sulfate, a key CNS disease biomarker, observed after three months of intravenous dosing Reductions in exploratory CSF biomarkers consistent with improved lysosomal function Reductions in levels of urine glycosaminoglycans following a switch from idursulfase support potential for improved peripheral effects relative to standard of care Generally well tolerated with safety […]
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