The DOI matches its Crossref record; 104 of the 104 references it cites were found in a registry or an index.
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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Cites
Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
Elbashir, S. M., Harborth, J., Lendeckel, W., Yalcin, A., Weber, K., & Tuschl, T. (2001) · Nature · doi:10.1038/35078107 · Cited by 9,456
Hannon, G. J. (2002) · Nature · doi:10.1038/418244a · Cited by 3,651
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
Adams, D., Gonzalez-Duarte, A., O’Riordan, W. D., Yang, C.-C., Ueda, M., Kristen, A. V., Tournev, I., Schmidt, H. H., Coelho, T., Berk, J. L., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1716153 · Cited by 3,047
Self-citationTafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy
Maurer, M. S., Schwartz, J. H., Gundapaneni, B., Elliott, P. M., Merlini, G., Waddington-Cruz, M., Kristen, A. V., Grogan, M., Witteles, R., Damy, T., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1805689 · Cited by 2,857
Self-citationProtein Misfolding, Amyloid Formation, and Human Disease: A Summary of Progress Over the Last Decade
Chiti, F., & Dobson, C. M. (2017) · Annual Review of Biochemistry · doi:10.1146/annurev-biochem-061516-045115 · Cited by 2,629
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
Benson, M. D., Waddington‐Cruz, M., Berk, J. L., Polydefkis, M. J., Dyck, P. J. B., Wang, A. K., Planté‐Bordeneuve, V., Barroso, F. A., Merlini, G., Obici, L. P., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1716793 · Cited by 1,483
Self-citationNatural History and Outcome in Systemic AA Amyloidosis
Lachmann, H. J., Goodman, H. J. B., Gilbertson, J. A., Gallimore, J. R., Sabin, C. A., Gillmore, J. D., & Hawkins, P. N. (2007) · New England Journal of Medicine · doi:10.1056/nejmoa070265 · Cited by 1,025
Palladini, G. O., Dispenzieri, A. A., Gertz, M. A., Kumar, S. K., Wechalekar, A. D., Hawkins, P. N., Schönland, S. O., Hegenbart, U., Comenzo, R. L., Kastritis, E., et al. (2012) · Journal of Clinical Oncology · doi:10.1200/jco.2011.37.7614 · Cited by 978
Safety and efficacy of RNAi therapy for transthyretin amyloidosis
Coelho, T., Adams, D., Silva, A., Lozeron, P., Hawkins, P. N., Mant, T., Perez, J., Chiesa, J., Warrington, S., Tranter, E., et al. (2013) · New England Journal of Medicine · doi:10.1056/nejmoa1208760 · Cited by 971
Tafamidis for transthyretin familial amyloid polyneuropathy
Coelho, T., Maia, L. F., SILVA, A. C. F. D., Waddington‐Cruz, M., Planté‐Bordeneuve, V., Lozeron, P., Suhr, O. B., Campistol, J. M., Conceição, I. M., Schmidt, H., et al. (2012) · Neurology · doi:10.1212/wnl.0b013e3182661eb1 · Cited by 812
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Research works printed but not linked: 6
- Quarta CC FalkRH SolomonSDet al. The prevalence of cardiac amyloidosis in familial amyloidotic polyneuropathy with predominant neuropathy: the diflunisal trial . XVth International Symposium on Amyloidosis (ISA) Indianapolis IN USA April 27–May 12014 .
- Obici L PerliniS PalladiniGet al. A Phase II study of doxycycline plus tauroursodeoxycholic acid in transthyretin amyloidosis . XIVth International Symposium on Amyloidosis (ISA) Uppsala Sweden ( 2016 ). Poster PA96 .
- Berk J , AdamsD, SuhrOet al. Long-term, open-label clinical experience with patisiran, an investigational RNAi therapeutic for patients with hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy . Orphanet. J. Rare Dis.12 ( Suppl. 1 ), 165 ( 2017 ).
- Solomon SD , AdamsD, KristenAet al. Effects of patisiran, an RNA interference therapeutic, on cardiac parameters in patients with hereditary transthyretin-mediated amyloidosis: an analysis of the APOLLO study . Circulation ( 2018 ) ( In press ).
- Suhr OB Gonzalez-DuarteA O’RiordanWet al. Long-term use of patisiran an investigational RNAi therapeutic in patients with hereditary transthyretin-mediated amyloidosis: baseline demographics and interim data form global open label extension study . Presented at: XVIth International Symposium On Amyloidosis (ISA) Kumamoto Japan . 26–29 March 2018 .
- Adams D , Gonzalez-DuarteA, O’RiordanWet al. Patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR amyloidosis with polyneuropathy: results from the Phase 3 APOLLO study . Orphanet. J. Rare Dis.12 ( Suppl. 1 ), 165 ( 2017 ).
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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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