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Long-term efficacy and safety of inotersen for hereditary transthyretin amyloidosis: NEURO-TTR open-label extension 3-year update.

Autor: Brannagan TH; Department of Neurology, Columbia University Medical Center, 710 West 168th Street, New York, NY, 10032, USA. tb2325@cumc.columbia.edu., Coelho T; Centro Hospitalar Universitário do Porto, Porto, Portugal., Wang AK; Neuromuscular Diagnostic Laboratory, University of California, Irvine, Orange, CA, USA., Polydefkis MJ; Department of Neurology, Johns Hopkins University, Baltimore, MD, USA., Dyck PJ; Neurology and General Internal Medicine, Mayo Clinic, Rochester, MN, USA., Berk JL; Amyloid Clinic, Boston University, Boston, MA, USA., Drachman B; Cardiovascular Medicine, Penn Presbyterian Medical Center, Philadelphia, PA, USA., Gorevic P; Department of Rheumatology, Mount Sinai Medical Center, New York, NY, USA., Whelan C; Cardiology, National Amyloidosis Centre, Royal Free Hospital, London, UK., Conceição I; Department of Neurology, CHULN, Hospital Santa Maria and Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal., Plante-Bordeneuve V; CHU Henri Mondor, Creteil, France., Merlini G; Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, University of Pavia, Pavia, Italy., Obici L; Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, University of Pavia, Pavia, Italy., Plana JMC; Hospital Clinic, University of Barcelona, Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain., Gamez J; Neurology Department, GMA Clinic, Autonomous University of Barcelona and European Reference Network on Rare Neuromuscular Diseases (ERN EURO-NMD), Barcelona, Spain., Kristen AV; Amyloidosis Center, Heidelberg University Hospital, Heidelberg, Germany., Mazzeo A; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Gentile L; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Narayana A; Akcea Therapeutics, Boston, MA, USA., Olugemo K; Akcea Therapeutics, Boston, MA, USA., Aquino P; Akcea Therapeutics, Boston, MA, USA., Benson MD; Department of Pathology and Laboratory Medicine, Indiana University School of Medicine, Indianapolis, IN, USA., Gertz M; Neurology and General Internal Medicine, Mayo Clinic, Rochester, MN, USA.

Publikováno v: Journal of neurology [J Neurol] 2022 Dec; Vol. 269 (12), pp. 6416-6427. Date of Electronic Publication: 2022 Jul 31.