Quality of life and effectiveness of vutrisiran as treatment for hereditary transthyretin amyloidosis

Although statistical significance was not reached, vutrisiran maintained or improved quality of life and clinical status in a real-world population that was more pretreated and had greater disease burden than the clinical trial. Combined with its favorable safety profile, subcutaneous administration, and extended dosing interval-preferred by patients-vutrisiran represents a promising therapeutic option for ATTRv amyloidosis. Long-term real-world studies are warranted to confirm these findings.