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Article

Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis

1 May 2019

Abstract excerpt

Hereditary transthyretin amyloidosis is a fatal autosomal dominant disorder characterized by deposition of transthyretin amyloid into the peripheral nervous system, heart, kidney, and gastrointestinal tract. Previous treatments using liver transplantation and small molecule stabilizers were not effective in stopping disease progression. Inotersen, a 2'-O-methyoxyethyl-modified antisense oligonucleotide, which...

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PMID: 31118583
DOI: 10.2147/dddt.s162913

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