Impact of Baseline Neuropathy Severity on Vutrisiran Treatment Response in the Phase 3 HELIOS-A Study

ConclusionsVutrisiran demonstrated benefit in neurologic function and other key efficacy measures versus external placebo across all four baseline neuropathy severity quartiles. Patients initiating vutrisiran earlier in their disease course retained the highest neurologic function level after 18  months, highlighting the importance of early diagnosis and treatment.Trial Registration NumberClinicalTrials.gov: NCT03759379.
Source: Neurology and Therapy - Category: Neurology Source Type: research