The data will be presented later today during a late-breaker session at WORLDSymposium. Denali management will host an analyst webinar today beginning at
An interim analysis included data on a total of five patients enrolled in Cohort A in the Phase 1/2 study, who all received three months of weekly intravenous doses of DNL310 after switching from idursulfase enzyme replacement therapy on Day 1 of the study. Key results included:
Normal levels of heparan sulfate, a glycosaminoglycan (GAG), in cerebrospinal fluid (CSF) that were seen after four weeks of dosing in four of five patients were sustained after three months of dosing (mean 85% reduction across Cohort A; p
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