Cited by 269Amyloidosis: Diagnosis, Treatment, OutcomesAlzheimer's disease research and treatmentsAmyotrophic Lateral Sclerosis Research

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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·2019·Neurodegenerative Disease Management

DOI 10.2217/nmt-2018-0033

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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.

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Authors: Kristen, A. V., Ajroud-Driss, S., Conceição, I., Gorevic, P., Kyriakides, T., & Obici, L.
Affiliations: Heidelberg University (DE)
Department of Neurology Northwestern University Feinberg School of Medicine Chicago IL 60611 USA
University of Lisbon (PT)
Mount Sinai Health System (US)
Cyprus Institute of Neurology and Genetics (CY)
Policlinico San Matteo Fondazione (IT)
ORCID: Kristen, Arnt V
Conceição, Isabel
Kyriakides, Theodoros
Obici, Laura
Venue: Neurodegenerative Disease Management
Publisher: Informa UK Limited
Published: 2018-11-27
Volume: 9
Issue: 1
Pages: 5-23
ISSN: 1758-2024 (ISSN-L)
1758-2032
DOI: 10.2217/nmt-2018-0033
OpenAlex: W2903444830
Cited by: 268
References: 104
Citations by year: 2026: 19 · 2025: 35 · 2024: 49 · 2023: 40 · 2022: 47 · 2021: 32 · 2020: 23 · 2019: 23
FWCI: 1.91
Type: journal article
Language: en
Open access: closed
Locations: Neurodegenerative Disease Management (publishedVersion)
PubMed (publishedVersion)
Topics: Health Sciences › Medicine › Internal Medicine › Amyloidosis: Diagnosis, Treatment, Outcomes
Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Alzheimer's disease research and treatments
Health Sciences › Medicine › Clinical Neurology › Amyotrophic Lateral Sclerosis Research
Keywords: patisiran, hereditary transthyretin amyloidosis, prealbumin, RNA interference, amyloidosis, neuropathy, cardiomyopathy, quality of life, drug efficacy, drug safety, clinical trials
MeSH terms: RNAi Therapeutics, Clinical Trials as Topic, Humans, Amyloid Neuropathies, Familial, RNA, Small Interfering

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Patisiran, an RNAi Therapeutic for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis

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