Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis

Arnt V. Kristen, Senda Ajroud-Driss, Isabel Conceição, Peter Gorevic, Theodoros Kyriakides, Laura Obici

Research output: Contribution to journalArticle

13 Citations (Scopus)

Abstract

Hereditary transthyretin-mediated amyloidosis is a rapidly progressive, heterogeneous disease caused by the accumulation of misfolded transthyretin protein as amyloid fibrils at multiple sites, and is characterized by peripheral sensorimotor neuropathy, autonomic neuropathy and/or cardiomyopathy. Current treatment options have limited efficacy and often do not prevent disease progression. Patisiran is a novel RNA interference therapeutic that specifically reduces production of both wild-type and mutant transthyretin protein. In Phase II, III and long-term extension studies in patients with hereditary transthyretin-mediated amyloidosis, patisiran has consistently slowed or improved progression of neuropathy. In addition, the Phase III trial demonstrated significant improvements in quality of life measures and indicators of cardiomyopathy. Here, we highlight efficacy and safety data from the patisiran clinical trial programme.

Original languageEnglish
Pages (from-to)5-23
Number of pages19
JournalNeurodegenerative disease management
Volume9
Issue number1
DOIs
Publication statusPublished - Feb 1 2019

Fingerprint

Prealbumin
Cardiomyopathies
Peripheral Nervous System Diseases
Mutant Proteins
RNA Interference
Amyloid
Disease Progression
Therapeutics
Quality of Life
Clinical Trials
Safety
patisiran
Amyloidosis, Hereditary, Transthyretin-Related
RNAi Therapeutics
Proteins

Keywords

  • cardiomyopathy
  • clinical trials
  • disease-modifying therapy
  • hATTR amyloidosis
  • patisiran
  • polyneuropathy

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis. / Kristen, Arnt V.; Ajroud-Driss, Senda; Conceição, Isabel; Gorevic, Peter; Kyriakides, Theodoros; Obici, Laura.

In: Neurodegenerative disease management, Vol. 9, No. 1, 01.02.2019, p. 5-23.

Research output: Contribution to journalArticle

Kristen, Arnt V. ; Ajroud-Driss, Senda ; Conceição, Isabel ; Gorevic, Peter ; Kyriakides, Theodoros ; Obici, Laura. / Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis. In: Neurodegenerative disease management. 2019 ; Vol. 9, No. 1. pp. 5-23.
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