Key Information
The content of this study was presented at the annual meeting of the American Academy of Neurology, May 2019, Philadelphia, Pennsylvania; the Fifth Congress of the European Academy of Neurology, June-July 2019, Oslo, Norway; the annual meeting of the Peripheral Nerve Society, June 2019, Genoa, Italy; and the annual meeting of the American Association of Neuromuscular & Electrodiagnostic Medicine, October 2019, Austin, Texas. Hereditary transthyretin-mediated amyloidosis (hATTR) is a rare, progressive, and fatal disease that results in the buildup of misfolded transthyretin (TTR) protein in major organ systems, ultimately leading to multisystem dysfunction and major organ failure. Hereditary ATTR is an autosomal-dominant disease most frequently caused by single nucleotide substitutions in the TTR gene.