Inotersen

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. Histor...

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Bibliographic Details
Published in:Neurodegenerative disease management 2019-02, Vol.9 (1), p.25-30
Main Authors: Benson, Merrill D, Dasgupta, Noel R, Monia, Brett P
Format: Article
Language:English
Subjects:
amyloidosis
inotersen
Tegsedi
transthyretin
Online Access:Get full text
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Description
Summary: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 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.
ISSN:1758-2024
1758-2032
DOI:10.2217/nmt-2018-0037