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An overview of drugs currently under investigation for the treatment of transthyretin-related hereditary amyloidosis

, MD & , MD

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Lidia Ciccone, Susanna Nencetti, Armando Rossello, Enrico Adriano Stura & Elisabetta Orlandini. (2016) Synthesis and structural analysis of halogen substituted fibril formation inhibitors of Human Transthyretin (TTR). Journal of Enzyme Inhibition and Medicinal Chemistry 31:sup1, pages 40-51.
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Francesca Lavatelli & Giampaolo Merlini. (2016) Advances in proteomic study of cardiac amyloidosis: progress and potential. Expert Review of Proteomics 13:11, pages 1017-1027.
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Fabian aus dem Siepen, Ralf Bauer, Matthias Aurich, Sebastian J Buss, Henning Steen, Klaus Altland, Hugo A Katus & Arnt V Kristen. (2015) Green tea extract as a treatment for patients with wild-type transthyretin amyloidosis: an observational study. Drug Design, Development and Therapy 9, pages 6319-6325.
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Giovanni Palladini, Paolo Milani & Giampaolo Merlini. (2015) Novel strategies for the diagnosis and treatment of cardiac amyloidosis. Expert Review of Cardiovascular Therapy 13:11, pages 1195-1211.
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Ming-Chang Chiang, Ming-Tsung Tseng, Chun-Liang Pan, Chi-Chao Chao & Sung-Tsang Hsieh. (2015) Progress in the treatment of small fiber peripheral neuropathy. Expert Review of Neurotherapeutics 15:3, pages 305-313.
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Articles from other publishers (37)

Julie Leann Han & Emilia Entcheva. (2023) Gene Modulation with CRISPR-based Tools in Human iPSC-Cardiomyocytes. Stem Cell Reviews and Reports 19:4, pages 886-905.
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Masamichi Inoue, Taishi Higashi, Yuya Hayashi, Risako Onodera, Kazuya Fujisawa, Toru Taharabaru, Ryoma Yokoyama, Kenta Ouchi, Yohei Misumi, Mitsuharu Ueda, Yasuteru Inoue, Mineyuki Mizuguchi, Takashi Saito, Takaomi C. Saido, Yukio Ando, Hidetoshi Arima, Keiichi Motoyama & Hirofumi Jono. (2022) Multifunctional Therapeutic Cyclodextrin-Appended Dendrimer Complex for Treatment of Systemic and Localized Amyloidosis. ACS Applied Materials & Interfaces 14:36, pages 40599-40611.
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Francesco Bemporad, Manuela Leri, Matteo Ramazzotti, Massimo Stefani & Monica Bucciantini. (2022) The Transthyretin/Oleuropein Aglycone Complex: A New Tool against TTR Amyloidosis. Pharmaceuticals 15:3, pages 277.
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Giorgia Cerqueni, Annachiara Scalzone, Caterina Licini, Piergiorgio Gentile & Monica Mattioli-Belmonte. (2021) Insights into oxidative stress in bone tissue and novel challenges for biomaterials. Materials Science and Engineering: C 130, pages 112433.
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Qi Qin, Cuibai Wei, YueShan Piao, Fang Lian, Hao Wu, Aihong Zhou, Fen Wang, Xiumei Zuo, Yue Han, Jihui Lyu, Dongmei Guo & Jianping Jia. (2021) Current Review of Leptomeningeal Amyloidosis Associated With Transthyretin Mutations. The Neurologist 26:5, pages 189-195.
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Filipa Bezerra, Maria João Saraiva & Maria Rosário Almeida. (2020) Modulation of the Mechanisms Driving Transthyretin Amyloidosis. Frontiers in Molecular Neuroscience 13.
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Kenneth W. Walker, Ian N. Foltz, Tina Wang, Hossein Salimi-Moosavi, Julie M. Bailis, Fei Lee, Phillip An, Stephen Smith, Richele Bruno & Zhulun Wang. (2020) The serum protein transthyretin as a platform for dimerization and tetramerization of antibodies and Fab fragments to enable target clustering. Journal of Biological Chemistry 295:30, pages 10446-10455.
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Joohee Lee, Kihyun Kim, Jin-Oh Choi, Seok Jin Kim, Eun-Seok Jeon & Joon Young Choi. (2020) 99mTc-DPD scintigraphy and SPECT/CT in patients with AL and ATTR type amyloidosis. Medicine 99:4, pages e18905.
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S. A. Boldueva, V. B. Petrova, V. Kh. Khavinson, M. V. Samohvalova & A. I. Petrova. (2020) Problems in the Diagnostics and Treatment of Transtiretinum Amyloidosis with Heart Disease in the Elderly: Clinical Experience. Advances in Gerontology 9:4, pages 466-471.
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Lorena Saelices, Binh A. Nguyen, Kevin Chung, Yifei Wang, Alfredo Ortega, Ji H. Lee, Teresa Coelho, Johan Bijzet, Merrill D. Benson & David S. Eisenberg. (2019) A pair of peptides inhibits seeding of the hormone transporter transthyretin into amyloid fibrils. Journal of Biological Chemistry 294:15, pages 6130-6141.
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Fredrick Nwude Eze, Ladda Leelawatwattana & Porntip Prapunpoj. (2019) Structural Stabilization of Human Transthyretin by Centella asiatica (L.) Urban Extract: Implications for TTR Amyloidosis. Biomolecules 9:4, pages 128.
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H. G. Zouari, S. Ng Wing Tin, A. Wahab, T. Damy & J.‐P. Lefaucheur. (2018) Assessment of autonomic innervation of the foot in familial amyloid polyneuropathy. European Journal of Neurology 26:1, pages 94.
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Arnt V. Kristen, Michael M. Kreusser, Patrick Blum, Stefan O. Schönland, Lutz Frankenstein, Andreas O. Dösch, Benjamin Knop, Matthias Helmschrott, Bastian Schmack, Arjang Ruhparwar, Ute Hegenbart, Hugo A. Katus & Philip W.J. Raake. (2018) Improved outcomes after heart transplantation for cardiac amyloidosis in the modern era. The Journal of Heart and Lung Transplantation 37:5, pages 611-618.
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Giorgina Barbara Piccoli, Mammar Hachemi, Ida Molfino, Jean Philippe Coindre & Charles Boursot. (2017) Doxycycline treatment in dialysis related amyloidosis: discrepancy between antalgic effect and inflammation, studied with FDG-positron emission tomography: a case report. BMC Nephrology 18:1.
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Tokio Matsuzaki, Yukio Akasaki, Merissa Olmer, Oscar Alvarez‐Garcia, Natalia Reixach, Joel N. Buxbaum & Martin K. Lotz. (2017) Transthyretin deposition promotes progression of osteoarthritis. Aging Cell 16:6, pages 1313-1322.
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Thomas P. Smith, Ian W. Windsor, Katrina T. Forest & Ronald T. Raines. (2017) Stilbene Boronic Acids Form a Covalent Bond with Human Transthyretin and Inhibit Its Aggregation. Journal of Medicinal Chemistry 60:18, pages 7820-7834.
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Stephen Connelly, David E. Mortenson, Sungwook Choi, Ian A. Wilson, Evan T. Powers, Jeffery W. Kelly & Steven M. Johnson. (2017) Semi-quantitative models for identifying potent and selective transthyretin amyloidogenesis inhibitors. Bioorganic & Medicinal Chemistry Letters 27:15, pages 3441-3449.
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Ana Gimeno, Luis M. Santos, Mobina Alemi, Josep Rivas, Daniel Blasi, Ellen Y. Cotrina, Jordi Llop, Gregorio Valencia, Isabel Cardoso, Jordi Quintana, Gemma Arsequell & Jesús Jiménez-Barbero. (2017) Insights on the Interaction between Transthyretin and Aβ in Solution. A Saturation Transfer Difference (STD) NMR Analysis of the Role of Iododiflunisal. Journal of Medicinal Chemistry 60:13, pages 5749-5758.
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Concetta Federico, Ketty Dugo, Francesca Bruno, Anna Maria Longo, Agata Grillo & Salvatore Saccone. (2017) Somatic mosaicism with reversion to normality of a mutated transthyretin allele related to a familial amyloidotic polyneuropathy. Human Genetics 136:7, pages 867-873.
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Xuan Chi, Philip Gatti & Thomas Papoian. (2017) Safety of antisense oligonucleotide and siRNA-based therapeutics. Drug Discovery Today 22:5, pages 823-833.
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Hartmut H.‐J. Schmidt, Fabio Barroso, Alejandra González‐Duarte, Isabel Conceição, Laura Obici, Denis Keohane & Leslie Amass. (2016) Management of asymptomatic gene carriers of transthyretin familial amyloid polyneuropathy. Muscle & Nerve 54:3, pages 353-360.
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Thibaud Damy, Bruno Costes, Albert A. Hagège, Erwan Donal, Jean-Christophe Eicher, Michel Slama, Aziz Guellich, Stéphane Rappeneau, Jean-Pierre Gueffet, Damien Logeart, Violaine Planté-Bordeneuve, Hélène Bouvaist, Olivier Huttin, Geneviève Mulak, Jean-Luc Dubois-Randé, Michel Goossens, Florence Canoui-Poitrine & Joel N. Buxbaum. (2016) Prevalence and clinical phenotype of hereditary transthyretin amyloid cardiomyopathy in patients with increased left ventricular wall thickness. European Heart Journal 37:23, pages 1826-1834.
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Massimo Russo, Gian Luca Vita, Claudia Stancanelli, Anna Mazzeo, Giuseppe Vita & Sonia Messina. (2016) Parenteral nutrition improves nutritional status, autonomic symptoms and quality of life in transthyretin amyloid polyneuropathy. Neuromuscular Disorders 26:6, pages 374-377.
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Manuela Leri, Daniele Nosi, Antonino Natalello, Riccardo Porcari, Matteo Ramazzotti, Fabrizio Chiti, Vittorio Bellotti, Silvia Maria Doglia, Massimo Stefani & Monica Bucciantini. (2016) The polyphenol Oleuropein aglycone hinders the growth of toxic transthyretin amyloid assemblies. The Journal of Nutritional Biochemistry 30, pages 153-166.
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Paola Florio, Claudia Folli, Michele Cianci, Daniele Del Rio, Giuseppe Zanotti & Rodolfo Berni. (2015) Transthyretin Binding Heterogeneity and Anti-amyloidogenic Activity of Natural Polyphenols and Their Metabolites. Journal of Biological Chemistry 290:50, pages 29769-29780.
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Takeshi Yokoyama, Shun Takaki, Keisuke Chosa, Takashi Sato, Mary Ann Suico, Yuriko Teranishi, Tsuyoshi Shuto, Mineyuki Mizuguchi & Hirofumi Kai. (2015) Structural stabilization of transthyretin by a new compound, 6-benzoyl-2-hydroxy-1H-benzo[de]isoquinoline-1,3(2H)-dione. Journal of Pharmacological Sciences 129:4, pages 240-243.
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Morie A. Gertz, Merrill D. Benson, Peter J. Dyck, Martha Grogan, Terresa Coelho, Marcia Cruz, John L. Berk, Violaine Plante-Bordeneuve, Hartmut H.J. Schmidt & Giampaolo Merlini. (2015) Diagnosis, Prognosis, and Therapy of Transthyretin Amyloidosis. Journal of the American College of Cardiology 66:21, pages 2451-2466.
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Márcia Waddington Cruz & Merril D. Benson. (2015) A Review of Tafamidis for the Treatment of Transthyretin-Related Amyloidosis. Neurology and Therapy 4:2, pages 61-79.
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Michele Cianci, Claudia Folli, Francesco Zonta, Paola Florio, Rodolfo Berni & Giuseppe Zanotti. (2015) Structural evidence for asymmetric ligand binding to transthyretin. Acta Crystallographica Section D Biological Crystallography 71:8, pages 1582-1592.
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Anna Mazzeo, Massimo Russo, Gianluca Di Bella, Fabio Minutoli, Claudia Stancanelli, Luca Gentile, Sergio Baldari, Scipione Carerj, Antonio Toscano & Giuseppe Vita. (2015) Transthyretin-Related Familial Amyloid Polyneuropathy (TTR-FAP): A Single-Center Experience in Sicily, an Italian Endemic Area. Journal of Neuromuscular Diseases 2:s2, pages S39-S48.
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John J. Chen, Joseph C. Genereux & R. Luke Wiseman. (2015) Endoplasmic reticulum quality control and systemic amyloid disease: Impacting protein stability from the inside out. IUBMB Life 67:6, pages 404-413.
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Traci L. Parry, Jason H. Melehani, Mark J. Ranek & Monte S. Willis. (2015) Functional Amyloid Signaling via the Inflammasome, Necrosome, and Signalosome: New Therapeutic Targets in Heart Failure. Frontiers in Cardiovascular Medicine 2.
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A. C. Martins, A. M. Rosa, E. Costa, C. Tavares, M. J. Quadrado & J. N. Murta. (2015) Ocular Manifestations and Therapeutic Options in Patients with Familial Amyloid Polyneuropathy: A Systematic Review. BioMed Research International 2015, pages 1-9.
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Jenny K W Lam, Michael Y T Chow, Yu Zhang & Susan W S Leung. (2015) siRNA Versus miRNA as Therapeutics for Gene Silencing. Molecular Therapy - Nucleic Acids 4, pages e252.
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Gian Maria Fabrizi & Giampietro Zanette. 2015. Prognosis of Neurological Diseases. Prognosis of Neurological Diseases 405 444 .
Maria M. Picken & Kevin Barton. 2015. Amyloid and Related Disorders. Amyloid and Related Disorders 489 505 .
Giorgia Cerqueni, Annachiara Scalzone, Caterina Licini, Piergiorgio Gentile & Monica Mattioli-Belmonte. (2020) Insights into Oxidative Stress in Bone Tissue and Novel Challenges for Biomaterials. SSRN Electronic Journal.
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