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.
Overview
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
In the citing paper
“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]
Citations
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
Self-citationRepurposing Diflunisal for Familial Amyloid Polyneuropathy
Berk, J. L., Suhr, O. B., Obici, L. P., Sekijima, Y., Zeldenrust, S. R., Yamashita, T., Heneghan, M. A., Gorevic, P. D., Litchy, W. J., Wiesman, J. F., et al. (2013) · JAMA · doi:10.1001/jama.2013.283815 · Cited by 719
Self-citationTransthyretin (TTR) Cardiac Amyloidosis
Ruberg, F. L., & Berk, J. L. (2012) · Circulation · doi:10.1161/circulationaha.111.078915 · Cited by 710
Hernández–Dı́az, S., & Rodríguez, L. A. G. (2000) · Archives of Internal Medicine · doi:10.1001/archinte.160.14.2093 · Cited by 707
Fibril in senile systemic amyloidosis is derived from normal transthyretin.
Westermark, P., Sletten, K., Johansson, B., & Cornwell, G. G. (1990) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.87.7.2843 · Cited by 698
Guideline of transthyretin-related hereditary amyloidosis for clinicians
Ando, Y., Coelho, T., Berk, J., Cruz, M., Ericzon, B., Ikeda, S., Lewis, W., Obici, L., Planté-Bordeneuve, V., Rapezzi, C., et al. (2013) · Orphanet Journal of Rare Diseases · doi:10.1186/1750-1172-8-31 · Cited by 682
Self-citationSipe, J. D., Benson, M. D., Buxbaum, J. N., Ikeda, S.-I., Merlini, G., Saraiva, M. J., & Westermark, P. (2016) · Amyloid · doi:10.1080/13506129.2016.1257986 · Cited by 603
Lipid nanoparticles enabling gene therapies: from concepts to clinical utility
Kulkarni, J. A., Cullis, P. R., & van der Meel, R. (2018) · Nucleic Acid Therapeutics · doi:10.1089/nat.2018.0721 · Cited by 592
Gillmore, J. D., Lovat, L. B., Persey, M. R., Pepys, M. B., & Hawkins, P. N. (2001) · The Lancet · doi:10.1016/s0140-6736(00)05252-1 · Cited by 586
Genotype and Phenotype of Transthyretin Cardiac Amyloidosis
Maurer, M. S., Hanna, M. A., Grogan, M. A., Dispenzieri, A. A., Witteles, R. M., Drachman, B. M., Judge, D. P., Lenihan, D. J., Gottlieb, S. S., Shah, S. J., et al. (2016) · Journal of the American College of Cardiology · doi:10.1016/j.jacc.2016.03.596 · Cited by 536
Self-citationSaraiva, M. J., Birken, S., Costa, P., & Goodman, D. S. (1984) · Journal of Clinical Investigation · doi:10.1172/jci111390 · Cited by 437
Lachmann, H. J., Gallimore, J. R., Gillmore, J. D., Carr-Smith, H. D., Bradwell, A. R., Pepys, M. B., & Hawkins, P. N. (2003) · British Journal of Haematology · doi:10.1046/j.1365-2141.2003.04433.x · Cited by 431
Transthyretin-Related Familial Amyloidotic Polyneuropathy
Ando, Y., Nakamura, M., & Araki, S. (2005) · Archives of Neurology · doi:10.1001/archneur.62.7.1057 · Cited by 416
Structure of a complex of two plasma proteins: transthyretin and retinol-binding protein
Monaco, H. L., Rizzi, M., & Coda, A. (1995) · Science · doi:10.1126/science.7754382 · Cited by 413
Glynn, L. E. (1981) · Immunology · Cited by 412
Holmgren, G., Steen, L., Ekstedt, J., Groth, C., Ericzon, B. ‐., Eriksson, S., Andersen, O., Karlberg, I. H., Nordén, G., Nakazato, M., et al. (1991) · Clinical Genetics · doi:10.1111/j.1399-0004.1991.tb03085.x · Cited by 395
Quarta, C. C., Solomon, S. D., Uraizee, I., Kruger, J., Longhi, S., Ferlito, M., Gagliardi, C., Milandri, A., Rapezzi, C., & Falk, R. H. (2014) · Circulation · doi:10.1161/circulationaha.113.006242 · Cited by 345
Coelho, T., Maurer, M. S., & Suhr, O. B. (2012) · Current Medical Research and Opinion · doi:10.1185/03007995.2012.754348 · Cited by 345
Rapezzi, C., Quarta, C. C., Obici, L. P., Perfetto, F., Longhi, S., Salvi, F., Biagini, E., Lorenzini, M., Grigioni, F., Leone, O., et al. (2012) · European Heart Journal · doi:10.1093/eurheartj/ehs123 · Cited by 330
Self-citationSuhr, O. B., Coelho, T., Buades, J., Pouget, J., Conceicao, I., Berk, J., Schmidt, H., Waddington-Cruz, M., Campistol, J. M., Bettencourt, B. R., et al. (2015) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-015-0326-6 · Cited by 321
Self-citationLiver Transplantation for Hereditary Transthyretin Amyloidosis
Ericzon, B., Wilczek, H., Larsson, M., Wijayatunga, P. D. C., Stangou, A. J., Peña, J., Furtado, E., Barroso, E., Daniel, J., Samuel, D., et al. (2015) · Transplantation · doi:10.1097/tp.0000000000000574 · Cited by 311
Online Registry for Mutations in Hereditary Amyloidosis Including Nomenclature Recommendations
Rowczenio, D. M., Noor, I., Gillmore, J. D., Lachmann, H. J., Whelan, C. J., Hawkins, P. N., Obici, L. P., Westermark, P., Grateau, G., & Wechalekar, A. D. (2014) · Human Mutation · doi:10.1002/humu.22619 · Cited by 307
Self-citation“Red‐flag” symptom clusters in transthyretin familial amyloid polyneuropathy
Conceição, I. M., González‐Duarte, A., Obici, L. P., Schmidt, H., Simoneau, D., Ong, M., & Amass, L. (2015) · Journal of the Peripheral Nervous System · doi:10.1111/jns.12153 · Cited by 298
Self-citationHerlenius, G., Wilczek, H., Larsson, M., & Ericzon, B. (2004) · Transplantation · doi:10.1097/01.tp.0000092307.98347.cb · Cited by 274
Huang, S. K., Mayhew, E. G., Gilani, S., Lasiç, D. D., Martin, F. J., & Papahadjopoulos, D. (1992) · PubMed · Cited by 274
Sekijima, Y., Dendle, M. A., & Kelly, J. W. (2006) · Amyloid · doi:10.1080/13506120600960882 · Cited by 269
Ruberg, F. L., Maurer, M. S., Judge, D. P., Zeldenrust, S. R., Skinner, M. M., Kim, A. Y., Falk, R. H., Cheung, K. N., Patel, A. R., Pano, A., et al. (2012) · American Heart Journal · doi:10.1016/j.ahj.2012.04.015 · Cited by 263
Castaño, A., Drachman, B. M., Judge, D. P., & Maurer, M. S. (2014) · Heart Failure Reviews · doi:10.1007/s10741-014-9462-7 · Cited by 258
Demonstration of transthyretin mRNA in the brain and other extrahepatic tissues in the rat.
Soprano, D. R., Herbert, J., Soprano, K. J., Schon, E. A., & Goodman, D. S. (1985) · Journal of Biological Chemistry · doi:10.1016/s0021-9258(17)39100-7 · Cited by 254
The course and prognostic factors of familial amyloid polyneuropathy after liver transplantation
Adams, D. (2000) · Brain · doi:10.1093/brain/123.7.1495 · Cited by 253
Evolving landscape in the management of transthyretin amyloidosis
Hawkins, P. N., Ando, Y., Dispenzeri, A., González‐Duarte, A., Adams, D., & Suhr, O. B. (2015) · Annals of Medicine · doi:10.3109/07853890.2015.1068949 · Cited by 248
Diagnostic pitfalls in sporadic transthyretin familial amyloid polyneuropathy (TTR-FAP)
Planté‐Bordeneuve, V., Ferreira, A. C., Lalu, T., Zaros, C., Lacroix, C., Adams, D., & Saïd, G. (2007) · Neurology · doi:10.1212/01.wnl.0000267338.45673.f4 · Cited by 243
Adams, D., Suhr, O. B., Hund, E., Obici, L. P., Tournev, I., Campistol, J. M., Slama, M., Hazenberg, B. P. C., & Coelho, T. (2016) · Current Opinion in Neurology · doi:10.1097/wco.0000000000000289 · Cited by 241
Self-citationSousa, A., Coelho, T., Barros, J., & Sequeiros, J. (1995) · American Journal of Medical Genetics · doi:10.1002/ajmg.1320600606 · Cited by 236
Sixty years of transthyretin familial amyloid polyneuropathy (TTR-FAP) in Europe
Parman, Y., Adams, D., Obici, L. P., Galán, L., Guergueltcheva, V., Suhr, O. B., & Coelho, T. (2016) · Current Opinion in Neurology · doi:10.1097/wco.0000000000000288 · Cited by 234
Self-citationCardiac amyloidosis: Updates in diagnosis and management
Mohty, D., Damy, T., Cosnay, P., Echahidi, N., Casset-Senon, D., Virot, P., & Jaccard, A. (2013) · Archives of cardiovascular diseases · doi:10.1016/j.acvd.2013.06.051 · Cited by 228
Koike, H., Tanaka, F., Hashimoto, R., Tomita, M., Kawagashira, Y., Iijima, M., Fujitake, J., Kawanami, T., Kato, T., Yamamoto, M., et al. (2012) · Journal of Neurology Neurosurgery & Psychiatry · doi:10.1136/jnnp-2011-301299 · Cited by 210
Transthyretin mutations in hyperthyroxinemia and amyloid diseases
Saraiva, M. J. (2001) · Human Mutation · doi:10.1002/humu.1132 · Cited by 206
Kelly, J. W. (1997) · Structure · doi:10.1016/s0969-2126(97)00215-3 · Cited by 205
Sattianayagam, P. T., Hahn, A. F., Whelan, C. J., Gibbs, S. D., Pinney, J. H., Stangou, A. J., Rowczenio, D. M., Pflugfelder, P. W., Fox, Z. V., Lachmann, H. J., et al. (2011) · European Heart Journal · doi:10.1093/eurheartj/ehr383 · Cited by 195
Transthyretin: A multifaceted protein
Vieira, M., & Saraiva, M. J. (2014) · BioMolecular Concepts · doi:10.1515/bmc-2013-0038 · Cited by 185
Cardoso, I., Martins, D. F., Ribeiro, T., Merlini, G., & Saraiva, M. J. (2010) · Journal of Translational Medicine · doi:10.1186/1479-5876-8-74 · Cited by 184
Adams, D., Suhr, O. B., Dyck, P. J. B., Litchy, W. J., Leahy, R. G., Chen, J., Gollob, J., & Coelho, T. (2017) · BMC Neurology · doi:10.1186/s12883-017-0948-5 · Cited by 181
Progression of transthyretin amyloid neuropathy after liver transplantation
Liepnieks, J. J., Zhang, L. Q., & Benson, M. D. (2010) · Neurology · doi:10.1212/wnl.0b013e3181ea15d4 · Cited by 158
Diflunisal for ATTR Cardiac Amyloidosis
Castaño, A., Helmke, S. T., Alvarez, J., Delisle, S., & Maurer, M. S. (2012) · Congestive Heart Failure · doi:10.1111/j.1751-7133.2012.00303.x · Cited by 156
Rapid progression of familial amyloidotic polyneuropathy
Adams, D., Coelho, T., Obici, L. P., Merlini, G., Mincheva, Z., Suanprasert, N., Bettencourt, B. R., Gollob, J., Gandhi, P. J., Litchy, W. J., et al. (2015) · Neurology · doi:10.1212/wnl.0000000000001870 · Cited by 155
Self-citationForty years of experience with type I amyloid neuropathy. Review of 483 cases
Coutinho, P., da Silva, A. L., Lopes, J. M., Resende, P.-E. A., Silva, A. M. D., Resende, L. A. L., Silva, A. M.-D., Filho, J. D. L., & Barbosa, A. (1980) · Cited by 149
Cardiac transthyretin amyloidosis
Dungu, J. N., Anderson, L. J., Whelan, C. J., & Hawkins, P. N. (2012) · Heart · doi:10.1136/heartjnl-2012-301924 · Cited by 140
Crooke, S. T. (2004) · Current Molecular Medicine · doi:10.2174/1566524043360375 · Cited by 139
Shin, S. C., & Robinson‐Papp, J. (2012) · Mount Sinai Journal of Medicine A Journal of Translational and Personalized Medicine · doi:10.1002/msj.21352 · Cited by 138
Gorevic, P. D., Prelli, F. C., Wright, J. R., Pras, M., & Frangione, B. (1989) · Journal of Clinical Investigation · doi:10.1172/jci113966 · Cited by 133
Self-citationSuhr, O. B., Lundgren, E., & Westermark, P. (2017) · Journal of Internal Medicine · doi:10.1111/joim.12585 · Cited by 133
Lozeron, P., Théaudin, M., Mincheva, Z., Ducot, B., Lacroix, C., Adams, D., & French Network for FAP (CORNAMYL) (2013) · European Journal of Neurology · doi:10.1111/ene.12225 · Cited by 129
Conceição, I. M., & de Carvalho, M. A. (2006) · Muscle & Nerve · doi:10.1002/mus.20644 · Cited by 128
Self-citationVinik, E. J., Vinik, A. I., Paulson, J. F., Merkies, I. S. J., Packman, J., Grogan, D. R., & Coelho, T. (2014) · Journal of the Peripheral Nervous System · doi:10.1111/jns5.12059 · Cited by 114
Wixner, J., Mundayat, R., Karayal, O. N., Anan, I., Karling, P., Suhr, O. B., & on behalf of the THAOS investigators (2014) · Orphanet Journal of Rare Diseases · doi:10.1186/1750-1172-9-61 · Cited by 112
Familial Amyloidosis: A Study of 52 North American-Born Patients Examined During a 30-Year Period
Gertz, M. A., Kyle, R. A., & Thibodeau, S. N. (1992) · Mayo Clinic Proceedings · doi:10.1016/s0025-6196(12)60388-7 · Cited by 112
Plasminogen activation triggers transthyretin amyloidogenesis in vitro
Mangione, P. P., Verona, G., Corazza, A., Marcoux, J., Canetti, D., Giorgetti, S., Raimondi, S., Stoppini, M., Esposito, M., Relini, A., et al. (2018) · Journal of Biological Chemistry · doi:10.1074/jbc.ra118.003990 · Cited by 111
Self-citationHereditary ATTR amyloidosis: a single-institution experience with 266 patients
Swiecicki, P. L., Zhen, D. B., Mauermann, M. L., Kyle, R. A., Zeldenrust, S. R., Grogan, M. A., Dispenzieri, A. A., & Gertz, M. A. (2015) · Amyloid · doi:10.3109/13506129.2015.1019610 · Cited by 110
Planté‐Bordeneuve, V., Gorram, F., Salhi, H., Nordine, T., Ayache, S. S., Corvoisier, P. L., Azoulay, D., Féray, C., Damy, T., & Lefaucheur, J. (2016) · Journal of Neurology · doi:10.1007/s00415-016-8337-3 · Cited by 108
Cerebral amyloid angiopathy in posttransplant patients with hereditary ATTR amyloidosis
Sekijima, Y., Yazaki, M., Oguchi, K., Ezawa, N., Yoshinaga, T., Yamada, M., Yahikozawa, H., Watanabe, M., Kametani, F., & Ikeda, S. (2016) · Neurology · doi:10.1212/wnl.0000000000003001 · Cited by 107
Recent advances in the treatment of familial amyloid polyneuropathy
Adams, D. (2012) · Therapeutic Advances in Neurological Disorders · doi:10.1177/1756285612470192 · Cited by 100
Cortese, A., Vita, G., Luigetti, M., Russo, M., Bisogni, G., Sabatelli, M., Manganelli, F., Santoro, L., Cavallaro, T., Fabrizi, G. M., et al. (2016) · Journal of Neurology · doi:10.1007/s00415-016-8064-9 · Cited by 98
Self-citationMatsuda, M., Gono, T., Morita, H., Katoh, N., Kodaira, M., & Ikeda, S. (2010) · European Journal of Neurology · doi:10.1111/j.1468-1331.2010.03215.x · Cited by 97
Hurst, J. W., Morris, D. C., & Alexander, R. W. (1999) · Clinical Cardiology · doi:10.1002/clc.4960220604 · Cited by 96
[Cardiac Transthyretin Amyloidosis].
Manganiello, C. F. (2020) · PubMed · Cited by 96
Novel conformation-specific monoclonal antibodies against amyloidogenic forms of transthyretin
Higaki, J. N., Chakrabartty, A., Galant, N. J., Hadley, K. C., Hammerson, B., Nijjar, T., Torres, R., Tapia, J. R., Salmans, J., Barbour, R., et al. (2016) · Amyloid · doi:10.3109/13506129.2016.1148025 · Cited by 92
Saïd, G., Grippon, S., & Kirkpatrick, P. N. (2012) · Nature Reviews Drug Discovery · doi:10.1038/nrd3675 · Cited by 86
Systemic amyloidosis: a challenge for the rheumatologist
Perfetto, F., Pignone, A. M., Livi, R., Tempestini, A., Bergesio, F., & Matucci‐Cerinic, M. (2010) · Nature Reviews Rheumatology · doi:10.1038/nrrheum.2010.84 · Cited by 86
Survival After Transplantation in Patients With Mutations Other Than Val30Met
Suhr, O. B., Larsson, M., Ericzon, B., & Wilczek, H. (2015) · Transplantation · doi:10.1097/tp.0000000000001021 · Cited by 76
FAP Neuropathy and Emerging Treatments
Adams, D., Théaudin, M., Cauquil, C., Algalarrondo, V., & Slama, M. (2014) · Current Neurology and Neuroscience Reports · doi:10.1007/s11910-013-0435-3 · Cited by 71
Impact of Liver Transplantation on Transthyretin-Related Ocular Amyloidosis in Japanese Patients
Hara, R. (2010) · Archives of Ophthalmology · doi:10.1001/archophthalmol.2009.390 · Cited by 71
Aboard transthyretin: From transport to cleavage
Liz, M. A., Mar, F. M., Franquinho, F., & Sousa, M. M. (2010) · IUBMB Life · doi:10.1002/iub.340 · Cited by 68
Tsuchiya, A., Yazaki, M., Kametani, F., Takei, Y., & Ikeda, S. (2008) · Liver Transplantation · doi:10.1002/lt.21395 · Cited by 67
Novel Drugs Targeting Transthyretin Amyloidosis
Hanna, M. (2014) · Current Heart Failure Reports · doi:10.1007/s11897-013-0182-4 · Cited by 65
Damy, T., Judge, D. P., Kristen, A. V., Berthet, K., Li, H., & Aarts, J. M. (2015) · Journal of Cardiovascular Translational Research · doi:10.1007/s12265-015-9613-9 · Cited by 64
Self-citationDe novo malignancies following liver transplantation: a case–control study with long‐term follow‐up
Yao, F. Y., Gautam, M., Palese, C., Rebres, R., Terrault, N. A., Roberts, J. P., & Peters, M. G. (2006) · Clinical Transplantation · doi:10.1111/j.1399-0012.2006.00527.x · Cited by 60
Ando, Y., Sekijima, Y., Obayashi, K., Yamashita, T., Ueda, M., Misumi, Y., Morita, H., Machii, K., Ohta, M., Takata, A., et al. (2016) · Journal of the Neurological Sciences · doi:10.1016/j.jns.2016.01.046 · Cited by 57
Pathogenesis of transthyretin amyloidosis
Benson, M. D. (2012) · Amyloid · doi:10.3109/13506129.2012.668501 · Cited by 40
Galant, N. J., Bugyei‐Twum, A., Rakhit, R., Walsh, P. J., Sharpe, S. J., Arslan, P. E., Westermark, P., Higaki, J. N., Torres, R., Tapia, J. R., et al. (2016) · Scientific Reports · doi:10.1038/srep25080 · Cited by 37
Assessment of nonsteroidal anti-inflammatory drug-induced cardiotoxicity
Singh, B. K., Haque, S. E., & Pillai, K. K. (2013) · Expert Opinion on Drug Metabolism & Toxicology · doi:10.1517/17425255.2014.856881 · Cited by 34
Klimtchuk, E. S., Prokaeva, T. B., Frame, N. M., Abdullahi, H., Spencer, B., Dasari, S., Cui, H., Berk, J. L., Kurtin, P. J., Connors, L. H., et al. (2018) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1802977115 · Cited by 34
New spastic paraplegia phenotype associated to mutation of NFU1
Tonduti, D., Dorboz, I., Imbard, A., Slama, A., Boutron, A., Pichard, S., Elmaleh, M., Vallée, L., Benoist, J., de Baulny, H. O., et al. (2015) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-015-0237-6 · Cited by 33
Drent, G., Graveland, C. W., Hazenberg, B. P. C., & Haagsma, E. B. (2009) · Amyloid · doi:10.1080/13506120903090726 · Cited by 29
Haagsma, E. B., Gameren, I. I. V., Bijzet, J., Posthumus, M. D., & Hazenberg, B. P. C. (2007) · Amyloid · doi:10.1080/13506120701461368 · Cited by 24
Newer Therapies for Amyloid Cardiomyopathy
Chakraborty, R., Muchtar, E., & Gertz, M. A. (2016) · Current Heart Failure Reports · doi:10.1007/s11897-016-0300-1 · Cited by 16
Cardiovascular Risk of NSAIDs: Time to Translate Knowledge into Practice
Reddy, K. S., & Roy, A. (2013) · PLoS Medicine · doi:10.1371/journal.pmed.1001389 · Cited by 16
Adams, D. H., & Karsten, V. (2019) · Revue Neurologique · doi:10.1016/j.neurol.2019.01.339 · Cited by 15
Adams, D., Coelho, T., Conceição, I. M., Waddington‐Cruz, M., Schmidt, H., Buades, J., Campistol, J. M., Pouget, J., Berk, J. L., Polydefkis, M. J., et al. (2017) · Neurology · doi:10.1212/wnl.88.16_supplement.s27.004 · Cited by 12
Self-citationAdams, D., Buades, J., Suhr, O. B., Obici, L. P., & Coelho, T. (2015) · Orphanet Journal of Rare Diseases · doi:10.1186/1750-1172-10-s1-p19 · Cited by 12
Self-citationAnalysis of disease progression in patients with transthyretin cardiac amyloidosis
Gilmore, J. D., Maurer, M. S., Vest, J., Karsten, V., Powell, C., Strahs, A. L., Gollob, J., & Hawkins, P. N. (2015) · Orphanet Journal of Rare Diseases · doi:10.1186/1750-1172-10-s1-o10 · Cited by 4
Conceição, I. M., Suhr, O. B., Coelho, T., Waddington‐Cruz, M., Schmidt, H., Buades, J., Campistol, J. M., Pouget, J., Berk, J. L., & Adams, D. (2016) · Neuromuscular Disorders · doi:10.1016/j.nmd.2016.06.206 · Cited by 3
Self-citationInfusion related reactions in patients with hATTR amyloidosis treated with patisiran
Polydefkis, M. J., Adams, D., Coelho, T., Kristen, A. V., González‐Duarte, A., Berk, J. L., Quan, D., Partisano, A. M., Gollob, J., Sweester, M., et al. (2018) · Journal of the Peripheral Nervous System · Cited by 2
Self-citationEfficiency of siRNA for removal of transthyretin V30M in a TTR leptomeningeal animal model
Gonçalves, P., Martins, S., Costelha, S., & Saraiva, M. J. (2017) · Amyloid · doi:10.1080/13506129.2016.1272452 · Cited by 2
Sekijima, Y., Tojo, K., Morita, H., Koyama, J., Koike, H., Sobue, G., & Ikeda, S. (2013) · Neurology · doi:10.1212/wnl.80.7_supplement.pd1.009 · Cited by 1
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 ).
Cited by
Sorted by citations
The Potential for microRNA Therapeutics and Clinical Research
Hanna, J., Hossain, G. S., & Kocerha, J. (2019) · Frontiers in Genetics · doi:10.3389/fgene.2019.00478 · Cited by 755
Advances in COVID-19 mRNA vaccine development
Fang, E., Liu, X., Li, M., Zhang, Z., Song, L., Zhu, B., Wu, X., Liu, J., Zhao, D., & Li, Y. (2022) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-022-00950-y · Cited by 601
Lipid Nanoparticles for Drug Delivery
Xu, L., Wang, X., Liu, Y., Yang, G., Falconer, R. J., & Zhao, C. (2021) · Advanced NanoBiomed Research · doi:10.1002/anbr.202100109 · Cited by 588
The Limitless Future of RNA Therapeutics
Damase, T. R., Sukhovershin, R. A., Boada, C., Taraballi, F., Pettigrew, R. I., & Cooke, J. P. (2021) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2021.628137 · Cited by 577
Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities
Tabrizi, S. J., Flower, M. D., Ross, C. A., & Wild, E. J. (2020) · Nature Reviews Neurology · doi:10.1038/s41582-020-0389-4 · Cited by 568
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
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review