![Sample our Bioscience journals, sign in here to start your access, Latest two full volumes FREE to you for 14 days]({"type":"image" , "src" : "/sda/5925/TOC-Subject-Sample-2021-Bioscience.jpg"})
Abstract
Hereditary transthyretin amyloidosis (ATTR) is a fatal systemic disease that results from deposition of the misfolded protein transthyretin (TTR) in tissues. Common clinical manifestations of ATTR include peripheral neuropathy, cardiomyopathy, autonomic dysfunction, diarrhea and constipation. Historically there have not been effective therapies for this devastating disease. Inotersen/Tegsedi™ (Akcea Therapeutics, MA, USA) is a second-generation antisense oligonucleotide (ASO) specific for TTR that inhibits production of TTR by the liver. In the recently completed Phase III NEURO-TTR study, inotersen was shown to be effective in stabilizing or improving peripheral neuropathy as measured by the modified neurologic impairment score +7 (mNIS+7) and improving the quality of life assessed by the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire. Inotersen is a breakthrough therapy for treatment of ATTR.
Keywords: :
Financial & competing interests disclosure
MD Benson and NR Dasgupta have received consulting fees from Ionis Pharmaceuticals. BP Monia works for Ionis Pharmaceuticals. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
No writing assistance was utilized in the production of this manuscript.