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Safety and Efficacy of Inotersen in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (NEURO-TTR) (N2.001)
Neurology2018Vol. 90(15_supplement)
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Morie A. Gertz, Annabel K. Wang, Teresa Coelho, Márcia Waddington‐Cruz, Michael Polydefkis, P. James B. Dyck, Morton Scheinberg, Violaine Planté‐Bordeneuve, John L. Berk, Fábio Barroso, David Adams, Thomas H. Brannagan, Carol Whelan, Giampaolo Merlini, Brian Drachman, Stephen B. Heitner, Isabel Conceição, Harmut Schmidt, Giuseppe Vita, Josep M. Campistol, Josep Gámez, Edward Gane, Peter D. Gorevic, Acary Souza Bullé Oliveira, Brett P. Monia, Steven G. Hughes, Jesse Kwoh, Bradley McEvoy, Brenda F. Baker, Elizabeth J. Ackermann, Merrill D. Benson
Abstract
To report the safety and efficacy of inotersen, an antisense oligonucleotide inhibitor of transthyretin (TTR) protein production, for the treatment of patients with hereditary TTR amyloidosis (hATTR) in a global, randomized, double-blind, placebo-controlled phase 3 study (NEURO-TTR, NCT01737398).
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