Inotersen (transthyretin-specific antisense oligonucleotide) for treatment of transthyretin amyloidosis

Author:

Benson Merrill D1,Dasgupta Noel R12,Monia Brett P3

Affiliation:

1. Department of Pathology & Laboratory Medicine, Indiana University, Indianapolis, IN, USA

2. Department of Medicine, Division of Cardiology, Indiana University, Indianapolis, IN, USA

3. Ionis Pharmaceuticals, Carlsbad, CA, USA

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.

Publisher

Future Medicine Ltd

Subject

Clinical Neurology

Reference30 articles.

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