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• W2904994225 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." @default.
• W2904994225 created "2018-12-22" @default.
• W2904994225 creator A5043229467 @default.
• W2904994225 creator A5044334898 @default.
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• W2904994225 date "2019-02-01" @default.
• W2904994225 modified "2023-09-24" @default.
• W2904994225 title "Inotersen (<i>transthyretin</i>-specific antisense oligonucleotide) for treatment of transthyretin amyloidosis" @default.
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• W2904994225 doi "https://doi.org/10.2217/nmt-2018-0037" @default.
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