Observational study evaluates quality of life improvements with vutrisiran in hereditary transthyretin amyloidosis, suggesting a beneficial treatment option.
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.
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Villanueva et al. (2026) studied this question.
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