Becoming familiar with hereditary transthyretin amyloidosis, a treatable neuropathy

Obici, Laura

doi:10.1590/0004-282x20180102

Cited by 1 publication

(1 citation statement)

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“…Transthyretin amyloidosis (ATTR) is an underdiagnosed, progressive, fatal multisystemic disease that results from the misfolding, aggregation, and deposition of the plasma transport protein, transthyretin (TTR), in various tissues through the body [1][2][3][4]. ATTR may be acquired, known as wild-type ATTR, or hereditary (ATTRv; v for variant, formerly abbreviated hATTR), which develops as a result of mutations in the TTR gene that destabilize the resultant TTR protein [1].…”

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confidence: 99%

Inotersen for the treatment of adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis

Gertz

Scheinberg

Waddington-Cruz

et al. 2019

Expert Review of Clinical Pharmacology

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Introduction: Hereditary transthyretin-mediated amyloidosis (ATTRv; v for variant) is an underdiagnosed, progressive, and fatal multisystemic disease with a heterogenous clinical phenotype that is caused by TTR gene mutations that destabilize the TTR protein, resulting in its misfolding, aggregation, and deposition in tissues throughout the body. Areas covered: Inotersen, an antisense oligonucleotide inhibitor, was recently approved in the United States and Europe for the treatment of the polyneuropathy of ATTRv based on the positive results obtained in the pivotal phase 3 trial, NEURO-TTR. This review will discuss the mechanism of action of inotersen and its pharmacology, clinical efficacy, and safety and tolerability. A PubMed search using the terms 'inotersen,' 'AG10,' 'antisense oligonucleotide,' 'hereditary transthyretin amyloidosis,' 'familial amyloid polyneuropathy,' and 'familial amyloid cardiomyopathy' was performed, and the results were screened for the most relevant English language publications. The bibliographies of all retrieved articles were manually searched to identify additional studies of relevance. Expert opinion: Inotersen targets the disease-forming protein, TTR, and has been shown to improve quality of life and neuropathy progression in patients with stage 1 or 2 ATTRv with polyneuropathy. Inotersen is well tolerated, with a manageable safety profile through regular monitoring for the development of glomerulonephritis or thrombocytopenia. ARTICLE HISTORYand wild-type TTR by targeting its mRNA. This article will review inotersen, an antisense oligonucleotide (ASO) inhibitor, for the treatment of polyneuropathy caused by ATTRv, focusing on the mechanism of action of inotersen and its pharmacology, clinical efficacy, and safety and tolerability.

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