Real-life experience with inotersen in hereditary transthyretin amyloidosis with late-onset phenotype: Data from an early-access program in Italy.
Marco LuigettiGiovanni AntoniniAndrea Di PaolantonioLuca GentileMarina GrandisLuca LeonardiAlessandro LozzaFiore ManganelliAnna MazzeoRoberta MussinelliFilomena MyLaura ObiciElena Maria PennisiViviana NocitiMassimo RussoMario SabatelliAlessandro SalvalaggioMatteo TagliapietraStefano TozzaPublished in: European journal of neurology (2022)
The long-term safety profile of inotersen is favorable. Neurologic disease severity at baseline is the main factor associated with progression.