Clinical EfficacyDNL310 demonstrated a 90% mean reduction in cerebrospinal fluid heparan sulfate from baseline, with sustained effects through Week 104, showcasing its strong potential in treating MPS II.
Regulatory ProgressDenali Therapeutics reached an agreement with the FDA for an accelerated approval path for DNL310, which is expected to significantly expedite the launch timeline for this promising treatment.
Safety And Biomarker DataRobust long-term safety and biomarker data supporting encouraging behavioral and cognitive improvements in treated patients further solidify the case for accelerated approval of DNL310.