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Papers of the Week

2022 Jul 07

Muscle Nerve

Long-Term Treatment Effects of Inotersen on Health-Related Quality of Life in Patients with hATTR Amyloidosis with Polyneuropathy: Analysis of the Open-Label Extension of the NEURO-TTR Trial.



Hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN) progressively affects patients' functionality and compromises health-related quality of life (HRQL). The aim of this study was to quantify the projected long-term treatment effects of inotersen versus placebo on HRQL measures.