Synopsis Inotersen improved the course of neurologic disease and quality of life in patients with hereditary transthyretin amyloidosis. The present study aims to determine the efficacy and safety of inotersen treatment in patients with hereditary transthyretin amyloidosis with polyneuropathy in the presence or absence of cardiomyopathy. This week’s “MED FACT” discusses a clinical trial from The New England Journal of Medicine, “Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis”. July 5, 2018. Authors: M.D.
Benson, M. Waddington ‑Cruz et. al., Key Takeaways Inotersen improved the course of neurologic disease and quality of life in patients with hereditary transthyretin amyloidosis. Objective: To determine the efficacy and safety of inotersen treatment in patients with hereditary transthyretin amyloidosis Study Type: International, randomized, double-blind,…
