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

Merrill Benson, Noel Dasgupta, Brett P. Monia

Research output: Contribution to journalArticle

4 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)25-30
Number of pages6
JournalNeurodegenerative disease management
Volume9
Issue number1
DOIs
StatePublished - Feb 1 2019

Fingerprint

Prealbumin
Antisense Oligonucleotides
Peripheral Nervous System Diseases
Familial Amyloidosis
Quality of Life
Diabetic Neuropathies
Constipation
Cardiomyopathies
Nervous System
Diarrhea
Therapeutics
Amyloidosis, Hereditary, Transthyretin-Related
Liver
Proteins

Keywords

  • amyloidosis
  • inotersen
  • Tegsedi™
  • transthyretin

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Inotersen (transthyretin-specific antisense oligonucleotide) for treatment of transthyretin amyloidosis. / Benson, Merrill; Dasgupta, Noel; Monia, Brett P.

In: Neurodegenerative disease management, Vol. 9, No. 1, 01.02.2019, p. 25-30.

Research output: Contribution to journalArticle

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