Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis

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 acts by reducing the production of transthyretin, was recently demonstrated to improve disease course and quality of life in early hereditary transthyretin amyloidosis polyneuropathy in a 15-month Phase III study.

• Publication year

  2019

• Venue

  Drug Design, Development and Therapy

• Publication date

  2019-05-01

• Fields of study

  Medicine

• Identifiers
  DOI 10.2147/DDDT.S162913PMID 31118583PMCID 6507904
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

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