Factors associated with increased health-related quality-of-life benefits in hereditary transthyretin amyloidosis polyneuropathy patients treated with inotersen.
Chafic KaramDuncan BrownMin YangNicolae DoneIbou DieyeAna BozasMontserrat Vera LlonchJames SignorovitchPublished in: Muscle & nerve (2022)
Patients who were younger and/or at earlier polyneuropathy stages experienced greater HRQL benefits from inotersen over 66 weeks. These findings underscore the need for early diagnosis and treatment initiation, especially among more severely affected patients in early stages of ATTRv-PN.