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Patisiran, an RNAi Therapeutic for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis
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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“For liver targets, lipid nanoparticles (LNPs) and N-acetylgalactosamine (GalNAc) conjugates are two clinically validated delivery strategies, with multiple regulatory approvals that include Onpattro® (patisiran) 2,3 , Givlaari® (givosiran) 4 , Oxlumo® (lumaisran) 5 , and Leqvio® (inclisiran) [6][7][8] , and numerous additional programs in various stages of clinical development across several thera”
From Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates (2022) · cites this paper as [3]
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Metadata
- 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)
- 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
- 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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