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FDA approves patisiran to treat hereditary transthyretin amyloidosis
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“In 2018, Patisiran (ONPATTRO™) gained its approval by the US FDA and became the first marketed RNAi-based therapy.”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [58]
Citations
Cites
Gene-silencing technology gets first drug approval after 20-year wait
Ledford, H. (2018) · Nature · doi:10.1038/d41586-018-05867-7 · Cited by 131
Treatment success in hereditary transthyretin amyloidosis
Fyfe, I. (2018) · Nature Reviews Neurology · doi:10.1038/s41582-018-0048-1 · Cited by 7
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“Patisiran is the first RNAi drug encapsulated in nanoparticle to be delivered into the liver cells, as they were approved by the FDA for the treatment of hereditary transthyretin amyloidosis [44, 45].”
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“Recently, two gene therapy drugs, inotersen and patisiran, were both approved for treating TTR-FAP (Keam 2018; Wood 2018).”
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“The clinical viability of nucleic acid-based therapies is further underscored by the FDA approval of patisiran, a small interfering RNA (siRNA) designed to target and degrade transthyretin mRNA for the management of rare polyneuropathy [35].”
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“The 2018 FDA approval of Onpattro (Adams et al. 2018; Wood 2018), an LNP-based siRNA formulation for the treat-ment of polyneuropathies caused by the hereditary disease transthyretin-mediated amyloidosis (hATTR), provided clinical validation of the LNP system for in vivo delivery of nucleic acid…”
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“To achieve intracellular delivery, Patisiran is formulated as a lipid nanoparticle drug using ionizable lipids [18].”
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“Data are mean ± SD, * P < 0.05, ** P < 0.01, and *** P < 0.001. ns, not significant [32, 33].”
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Metadata
- Authors
- Wood, H.
- Corresponding author
- Wood, Heather
- ORCID
- Wood, Heather
- Venue
- Nature Reviews Neurology
- Published
- 2018-08-29
- Volume
- 14
- Issue
- 10
- Pages
- 570-570
- ISSN
- 1759-4758 (ISSN-L)
- 1759-4766
- OpenAlex
- W2888899523
- Cited by
- 145
- References
- 2
- Citations by year
- 2026: 9 · 2025: 12 · 2024: 12 · 2023: 22 · 2022: 14 · 2021: 23 · 2020: 20 · 2019: 28 · 2018: 5
- FWCI
- 6.64
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- Locations
- Nature Reviews Neurology (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, RNA interference therapy, prealbumin, FDA drug approval, rare genetic disorders, antisense oligonucleotides, protein misfolding
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