Citations (161)
Keep up to date with the latest research on this topic with citation updates for this article.
Read on this site (24)
Simina Ticau, Emre Aldinc, Michael Polydefkis, David Adams, Teresa Coelho, Mitsuharu Ueda, Cecilia Hale, John Vest & Paul Nioi. (2023) Treatment response and neurofilament light chain levels with long-term patisiran in hereditary transthyretin-mediated amyloidosis with polyneuropathy: 24-month results of an open-label extension study. Amyloid 0:0, pages 1-11.
Read now
Read now
Madeline Merkel, David Danese, Chongshu Chen, Jessie Wang, Aozhou Wu, Hongbo Yang & Hollis Lin. (2023) Indirect treatment comparison (ITC) of the efficacy of vutrisiran and tafamidis for hereditary transthyretin-mediated amyloidosis with polyneuropathy. Expert Opinion on Pharmacotherapy 24:10, pages 1205-1214.
Read now
Read now
Meng Jiang, Mengdie Wang, Zhengyu Tao, Yezi Chai, Qiming Liu, Qifan Lu, Qizhen Wu, Xiaoying Ying, Yanan Huang, Ying Nie, Yuqi Tang, Xin Zhang, Yu Liu & Jun Pu. (2023) Biochemical and biophysical properties of an unreported T96R mutation causing transthyretin cardiac amyloidosis. Amyloid 30:2, pages 188-198.
Read now
Read now
Dianna Quan, Laura Obici, John L. Berk, Yukio Ando, Emre Aldinc, Matthew T. White & David Adams. (2023) Impact of baseline polyneuropathy severity on patisiran treatment outcomes in the APOLLO trial. Amyloid 30:1, pages 49-58.
Read now
Read now
David Adams, Ivailo L. Tournev, Mark S. Taylor, Teresa Coelho, Violaine Planté-Bordeneuve, John L. Berk, Alejandra González-Duarte, Julian D. Gillmore, Soon-Chai Low, Yoshiki Sekijima, Laura Obici, Chongshu Chen, Prajakta Badri, Seth M. Arum, John Vest & Michael Polydefkis. (2023) Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. Amyloid 30:1, pages 18-26.
Read now
Read now
Sumit Gangopadhyay & Kiran R Gore. (2022) Advances in siRNA therapeutics and synergistic effect on siRNA activity using emerging dual ribose modifications. RNA Biology 19:1, pages 452-467.
Read now
Read now
Teresa Coelho, Isabel Conceição, Márcia Waddington-Cruz, Denis Keohane, Marla B. Sultan, Doug Chapman & Leslie Amass. (2022) A natural history analysis of asymptomatic TTR gene carriers as they develop symptomatic transthyretin amyloidosis in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Amyloid 29:4, pages 228-236.
Read now
Read now
Yukio Ando, David Adams, Merrill D. Benson, John L. Berk, Violaine Planté-Bordeneuve, Teresa Coelho, Isabel Conceição, Bo-Göran Ericzon, Laura Obici, Claudio Rapezzi, Yoshiki Sekijima, Mitsuharu Ueda, Giovanni Palladini & Giampaolo Merlini. (2022) Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis. Amyloid 29:3, pages 143-155.
Read now
Read now
Brian Tomlinson, Elaine Chow, Paul Chan & Christopher Wai Kei Lam. (2021) An evaluation of the pharmacokinetics of inclisiran in the treatment of atherosclerotic cardiovascular disease. Expert Opinion on Drug Metabolism & Toxicology 17:12, pages 1353-1361.
Read now
Read now
Peter Gorevic, Jaclyn Franklin, Jihong Chen, Gautam Sajeev, Jessie C. H. Wang & Hollis Lin. (2021) Indirect treatment comparison of the efficacy of patisiran and inotersen for hereditary transthyretin-mediated amyloidosis with polyneuropathy. Expert Opinion on Pharmacotherapy 22:1, pages 121-129.
Read now
Read now
Marco Luigetti, Angela Romano, Andrea Di Paolantonio, Giulia Bisogni & Mario Sabatelli. (2020) Diagnosis and Treatment of Hereditary Transthyretin Amyloidosis (hATTR) Polyneuropathy: Current Perspectives on Improving Patient Care. Therapeutics and Clinical Risk Management 16, pages 109-123.
Read now
Read now
Ola Akinboboye, Keyur Shah, Alberta L. Warner, Thibaud Damy, Herman A. Taylor, Jared Gollob, Christine Powell, Verena Karsten, John Vest & Mathew S. Maurer. (2020) DISCOVERY: prevalence of transthyretin (TTR) mutations in a US-centric patient population suspected of having cardiac amyloidosis. Amyloid 27:4, pages 223-230.
Read now
Read now
Margaret M. Parker, Simina Ticau, James Butler, David Erbe, Madeline Merkel, Emre Aldinc, Gregory Hinkle & Paul Nioi. (2020) Transthyretin-stabilising mutation T119M is not associated with protection against vascular disease or death in the UK Biobank. Amyloid 27:3, pages 184-190.
Read now
Read now
Laura Obici, John L. Berk, Alejandra González-Duarte, Teresa Coelho, Julian Gillmore, Hartmut H.-J. Schmidt, Matthias Schilling, Taro Yamashita, Céline Labeyrie, Thomas H. Brannagan$suffix/text()$suffix/text(), Senda Ajroud-Driss, Peter Gorevic, Arnt V. Kristen, Jaclyn Franklin, Jihong Chen, Marianne T. Sweetser, Jing Jing Wang & David Adams. (2020) Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis. Amyloid 27:3, pages 153-162.
Read now
Read now
Luis F. Maia, Aleksandra Maceski, Isabel Conceição, Laura Obici, Rui Magalhães, Andreas Cortese, David Leppert, Giampaolo Merlini, Jens Kuhle & Maria João Saraiva. (2020) Plasma neurofilament light chain: an early biomarker for hereditary ATTR amyloid polyneuropathy. Amyloid 27:2, pages 97-102.
Read now
Read now
Veena Mathew & Annabel K Wang. (2019) Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis. Drug Design, Development and Therapy 13, pages 1515-1525.
Read now
Read now
Paolo Milani, Roberta Mussinelli, Stefano Perlini, Giovanni Palladini & Laura Obici. (2019) An evaluation of patisiran: a viable treatment option for transthyretin-related hereditary amyloidosis. Expert Opinion on Pharmacotherapy 20:18, pages 2223-2228.
Read now
Read now
Morie A. Gertz, Morton Scheinberg, Márcia Waddington-Cruz, Stephen B. Heitner, Chafic Karam, Brian Drachman, Sami Khella, Carol Whelan & Laura Obici. (2019) Inotersen for the treatment of adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis. Expert Review of Clinical Pharmacology 12:8, pages 701-711.
Read now
Read now
Isabel Conceição, Teresa Coelho, Claudio Rapezzi, Yeşim Parman, Laura Obici, Lucía Galán & Antoine Rousseau. (2019) Assessment of patients with hereditary transthyretin amyloidosis – understanding the impact of management and disease progression. Amyloid 26:3, pages 103-111.
Read now
Read now
Violaine Planté-Bordeneuve, Hollis Lin, Jared Gollob, Sonalee Agarwal, Marissa Betts, Kyle Fahrbach, Madhura Chitnis & Michael Polydefkis. (2019) An indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy. Expert Opinion on Pharmacotherapy 20:4, pages 473-481.
Read now
Read now
Junyi Yang. (2019) Patisiran for the treatment of hereditary transthyretin-mediated amyloidosis. Expert Review of Clinical Pharmacology 12:2, pages 95-99.
Read now
Read now
Mario Nuvolone & Giampaolo Merlini. (2017) Emerging therapeutic targets currently under investigation for the treatment of systemic amyloidosis. Expert Opinion on Therapeutic Targets 21:12, pages 1095-1110.
Read now
Read now
Hartmut Schmidt, Márcia Waddington Cruz, Marc F. Botteman, John A. Carter, Avijeet Chopra, Michelle Stewart, Markay Hopps, Shari Fallet & Leslie Amass. (2017) Global epidemiology of transthyretin hereditary amyloid polyneuropathy: a systematic review. Amyloid 24:sup1, pages 111-112.
Read now
Read now
David Adams, Cecile Cauquil, Céline Labeyrie, Guillemette Beaudonnet, Vincent Algalarrondo & Marie Théaudin. (2016) TTR kinetic stabilizers and TTR gene silencing: a new era in therapy for familial amyloidotic polyneuropathies. Expert Opinion on Pharmacotherapy 17:6, pages 791-802.
Read now
Read now
Articles from other publishers (137)
Juan Zhou, Yanfang LiJing GengHong ZhouLian LiuXiaochun Peng. (2023) Recent Progress in the Development and Clinical Application of New Drugs for Transthyretin Cardiac Amyloidosis. Journal of Cardiovascular Pharmacology 82:6, pages 427-437.
Crossref
Crossref
Shion A. Lim, Nicholas J. Agard, Ryan L. Kelly, Benjamin N. Bell, Si-Jie Chen, Anna M. Gram, J. Robert MerrittIan MoenchKatarina PanceTatjana Petojevic. 2023. 2023 Medicinal Chemistry Reviews. 2023 Medicinal Chemistry Reviews
713
798
.
Carmen Tsang, Ahsan Huda, Max Norman, Carissa Dickerson, Vincenzo Leo, Jack Brownrigg, Mamas Mamas & Perry Elliott. (2023) Detecting transthyretin amyloid cardiomyopathy (ATTR-CM) using machine learning: an evaluation of the performance of an algorithm in a UK setting. BMJ Open 13:10, pages e070028.
Crossref
Crossref
Laura Obici, Senda Ajroud-Driss, Kon-Ping Lin, John L. Berk, Julian D. Gillmore, Parag Kale, Haruki Koike, David Danese, Emre Aldinc, Chongshu Chen, John Vest, David Adams, Jonas Wixner, Rolf Backlund, Björn Pilebro, Intissar Anan, Fredrik Edbom, Anna Ekman, Sandra Arvidsson, Ulrika Englund, Karin Söderberg, Erik Nordh, Erica Uneus, Kristin Samuelsson, Anna Nilzen, Rayomand Press, Mirjam Bilecen, Teresa Coelho, Marta Novais, Patricia Rodrigues, Ana Martins da Silva, Inês Cardoso, Carla Rodrigues, Joana Ramalho, Helder Martins, Mónica Silva, Nádia Guimaraes, Javier Perez, Antonio Hipólito Reis, Julia Monte, Natalia Ferreira, Cristina Alves, Marcio Cardoso, Ricardo Teixeira, Isabel Conceição, Filipa Lamas, Miguel Oliveira Santos, Catarina Campos, Conceiçao de Azevedo Coutinho, José Castro, Isabel Castro, Daniela Silva, Susana Gonçalves, Eleonora Di Buduo, Claudia Sforzini, Roberta Mussinelli, Vittorio Rosti, Alessandro Lozza, Anna Racchi, Mario Sabatelli, Marco Luigetti, Giulia Bisogni, Angela Romano, Valeria Guglielmino, Andrea Di Paolantonio, Daniela Bernardo, Giuseppe Vita, Anna Mazzeo, Massimo Russo, Davide Pareyson, Daniela Calabrese, Silvia Fenu, Paola Saveri, Hans Nienhuis, Geert Bokhorst, Carlien Roos, Margriet Couperus, Greetje De Jong, Anne Brunger, Gea Drost, Fiete Lange, Adinda Colauto, Márcia Waddington-Cruz, Aline Abreu, Roberto Coury Pedrosa, Renata Gervais de Santa Rosa, Moisés Dias, Fetra Rakotondratafika, Andoni Echaniz-Laguna, Cecile Cauquil, Céline Labeyrie, Guillemette Beaudonnet, Yasmine Boubrit, Amina Gaouar, Halima Bourenane, Shahram Attarian, El Khansa Yahia, Annie Verschueren, Aude-Marie Grapperon, Emilien Delmont, Violaine Planté-Bordeneuve, Laetitia Vervoitte, Samar S. Ayache, Philippe Le Corvoisier, Raphaele Arrouasse, Thierry Gendre, Laure Abou Chakra, Cécile Focsénéanu, Caroline Barau, Guilhem Sole, Laurie Belin, Marie Helene Violleau, Fanny Duval-Bontemps, Rami Massie, Xin Dong, Francisco Muñoz-Beamud, Sandra García Garrido, Cristina Borrachero, Alvaro Gragera Martinez, Lucía Galán Dávila, Marta Palacios, Laura M. Vicente, Leopoldo Perez de Isla, Carlos Casasnovas, Carles Díez López, Elena Fabra, José González-Costello, Sonia Guerrero, Sergi Yun Viladomat, Yurema Martinez, Valentina Velez-Santamaria, Velina Nedkova-Hristova, Pablo Garcia Pavia, Ariadna Gonzalez Segovia, Fernando De Frutos, Esther Gonzalez-Lopez, Fernando Dominguez, Luis E. Escobar-López, Eva Cabrera-Romero, Paula Sánchez Gismera, María de la Iglesia, Fernando Martinez Valle, Gonzalo Mazuela Aguila, Karen Lorite, Núria Raguer, Pilar Suñé, Pablo Piera, Carlos Ortega, Carla Aguilar, Gisela Gili, Hartmut Schmidt, Christel Langenstroer, Anna Hüsing-Kabar, Iyad Kabar, Matthias Schilling, Frauke Friebel, Phil-Robin Tepasse, Frank Birklein, Monika Firros, Fabiola Escolano-Lozano, Caitlin Brueckner, Vanessa Bahnam, Michelle C. Kaku, K. H. Vincent Lau, Janice Wiesman, Martha Grogan, Susanna Miller, Janell Frantz, Diane C. Schmidt, Omar AbouEzzeddine, Wayne Miller, Grace Lin, Morie Gertz, Angela Dispenzieri, Thomas Brannagan, Raisy Fayerman, Elizabeth DuVerger, Jorge Cabrera, Mathew S. Maurer, Christina M. Ulane, Louis H. Weimer, Stephen Tsang, Jeffrey Shije, Nathan Carberry, Sai Si Thu, Dianna Quan, Brianna Blume, J. Scott Overcash, He Pu, Kia Lee, Hanh Chu, Karla Zepeda, Michael Waters, Thao Vuong, Derya Coskun, Kimberly Quillin, Allison Davis, Michael Polydefkis, Jing Ye, Xiaoling Li, Mohammad Khoshnoodi, Geno Vista, Tae Hwan Chung, Michele Watt, Dan Tsottles, Ahmad Masri, Dayna Carlson, Brian Drachman, Patricia Divito, Hansie Mathelier, Margaret Shanks, Karen Maslowski, Sami Khella, Janice Pieretti, Benjamin Joslin, Emma Schmidt, Miriam Freimer, Julie Agriesti, Fabio Barroso, Florencia Picone, Andrea Lautre, Lucas Orellana, Wenqin Du, Joost Felius, Alejandra González-Duarte, Karla Cardenas Soto, Rebecca Traub, Manisha Chopra, Chi-Chao Chao, Chia-Hua Hsu, Li-Kai Tsai, Ming-Jen Lee, Jen-Jen Su, Sung-Tsang Hsieh, Hsueh-Wen Hsueh, Hsi-Chieh Chou, Byoung-Joon Kim, Hyesun Kang, Ju-Hong Min, Eun-Seok Jeon, Yeon Hak Chung, Jae Hong Park, Jeeyoung Oh, Hyun Joo Jeong, Ivailo Tournev, Sashka Zhelyazkova, Yohei Misumi, Yumiko Sakamoto, Nami Hashimoto, Yoshimi Misumi, Aya Takahashi, Mitsuharu Ueda, Teruaki Masuda, Akihiko Ueda, Masahisa Katsuno, Kazuki Tajima, Momoko Sumi, Fujiko Hasegawa, Takahiro Okumura, Masahiro Iijima, Yuki Fukami, Daisuke Ito, Yoshiyuki Kishimoto, Tomoyuki Kazuta, Katsuhiko Kato, Naohiro Mouri, Soma Furukawa, Ryoji Nishi, Yoshiki Sekijima, Keiko Ito, Nagaaki Kato, Dai Kishida, Hideki Mochizuki, Kaori Okada, Kurumi Ohashi, Kensuke Ikenaka, Masayuki Nakamori, Makoto Kinoshita, Bella Ruth Mapalo, Steven Law, Liza Chacko, Helen Lachmann, Oliver Cohen, Yousuf Siu Kay Razvi, Sindhu Varughese, Ana Martinez-Naharro, Richard Orrell, Marianna Fontana, Lisa Rannigan, Sarah Louth, Eleni Zamba-Papanicolaou, Demetra Charalamnibous, Rana Abu Manneh, Kleopas Kleopa, Theodoros Christodoulides, Savvas Frangos, Michele Galganski-Cleanthous, Eftychia Gaglia, Irene Smoleski, Elena Kkolou, Andry Ploutarchou, Mariana Hanghiuc, Galini Chroidou, Olga Stylianou, Anastasia Krokou, Irene Zannetou, Efstathios Kastritis, Dimitra Papadopoulou, Ilias Spinasas, Panayiotis Bakalis, Nikolaos Kanellias, Despoina Fotiou, Ioanna Dialoupi, Magdalini Migko, Maria Gavriatopoulou, Soon-Chai Low, Mark Taylor, Graeme Stewart, Helen Knight, Steve Vucic, Antonia Carroll, Matthew Silsby, Dan Suan, Simon Gibbs, Carmela Corfield, Suzana Jakicic, Hayden Jina, Stephen Ting, Shi Qin Wong, Peter Mollee, Lynda McKinley, Emad Abro, Dariusz Korczyk, Gauthier Remiche, Nick Alaerts, Fabienne De Veylder, Kristl Claeys, Elisa Debien, Joyce Cremers, Ann D’hondt & Bram De Wel. (2023) Impact of Vutrisiran on Quality of Life and Physical Function in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy. Neurology and Therapy 12:5, pages 1759-1775.
Crossref
Crossref
Jing-Yi Zhang, Ya-Tao Wang, Lu Sun, Sai-Qi Wang & Zhe-Sheng Chen. (2023) Synthesis and clinical application of new drugs approved by FDA in 2022. Molecular Biomedicine 4:1.
Crossref
Crossref
Mengli Wang, Honglan Yang, Zhiqiang Lin, Xiaobo Li, Lei Liu, Shunxiang Huang, Huadong Zhao, Xiying Zhu, Qiao Xiao, Ranhui Duan, Junling Wang, Stephan Zuchner, Beisha Tang & Ruxu Zhang. (2023) The genetic and clinical spectrum in a cohort of 39 families with complex inherited peripheral neuropathies. Journal of Neurology.
Crossref
Crossref
Marina Stavrou, Alexia Kagiava, Irene Sargiannidou, Elena Georgiou & Kleopas A. Kleopa. (2023)
Charcot–Marie–Tooth
neuropathies: Current gene therapy advances and the route toward translation
. Journal of the Peripheral Nervous System 28:2, pages 150-168.
Crossref
Crossref
Jan L. De Bleecker, Kristl G. Claeys, Stéphanie Delstanche, Vinciane Van Parys, Jonathan Baets, Sébastien Tilleux & Gauthier Remiche. (2023) A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium. Acta Neurologica Belgica 123:3, pages 1029-1037.
Crossref
Crossref
Efstratios A. Apostolou, Anna-Maria Fontrier, Georgios K. Efthimiadis, Efstathios Kastritis, John Parissis & Panos Kanavos. (2023) The patient pathway in ATTR-CM in Greece and how to improve it: A multidisciplinary perspective. Hellenic Journal of Cardiology.
Crossref
Crossref
Sachin Kumar, Vijay Kumar Bhardwaj, Rahul Singh & Rituraj Purohit. (2023) Structure restoration and aggregate inhibition of V30M mutant transthyretin protein by potential quinoline molecules. International Journal of Biological Macromolecules 231, pages 123318.
Crossref
Crossref
Zhenyue Fu, Jiayu Lv, Xiya Gao, Bingxuan Zhang, Yumeng Li, Xia Xu, Haoran Zheng, Huaqin Wu & Qingqiao Song. (2023) Research trends and hotspots evolution of cardiac amyloidosis: a bibliometric analysis from 2000 to 2022. European Journal of Medical Research 28:1.
Crossref
Crossref
Christopher Robinson, Cynthia Pham, Alec M. Zamarripa, Chase S. Dugay, Christopher A. Lee, Amnon A. Berger, Avi Landman, Elyse M. Cornett, Hisham Kassem, Alan D. Kaye, Ivan Urits, Omar Viswanath & Latha Ganti. (2023) Inotersen to Treat Polyneuropathy Associated with Hereditary Transthyretin (hATTR) Amyloidosis. Health Psychology Research 10:5.
Crossref
Crossref
John P. Cooke & Keith A. Youker. (2022) Future Impact of mRNA Therapy on Cardiovascular Diseases. Methodist DeBakey Cardiovascular Journal 18:5, pages 64-73.
Crossref
Crossref
Maria M. Picken. (2022) Current Understanding of Systemic Amyloidosis and Underlying Disease Mechanisms. The American Journal of Cardiology 185, pages S2-S10.
Crossref
Crossref
Sandrine Lemoine & Marie Courbebaisse. (2022) Petits ARN interférents : applications potentielles pour les néphrologues Small interfering RNA: potential applications for nephrologists. Néphrologie & Thérapeutique 18:6, pages 6S1-6S6.
Crossref
Crossref
Lucy Lee, Rakesh Gollen, Anas M. Fathallah, Lan Gao & Shivakumar Patil. (2022) Bridging the Gap With Clinical Pharmacology in Innovative Rare Disease Treatment Modalities: Targeting DNA to RNA to Protein. The Journal of Clinical Pharmacology 62:S2.
Crossref
Crossref
Ruo‐Jing Li, Lian Ma, Fang Li, Liang Li, Youwei Bi, Ye Yuan, Yangbing Li, Yuan Xu, Xinyuan Zhang, Jiang Liu, Venkatesh Atul Bhattaram, Jie Wang, Robert Schuck, Michael Pacanowski & Hao Zhu. (2022) Model‐Informed Approach Supporting Drug Development and Regulatory Evaluation for Rare Diseases. The Journal of Clinical Pharmacology 62:S2.
Crossref
Crossref
Martina Schmidt, Ali Yilmaz, Michael Bietenbeck, Matthias Schilling, Christoph Röcken & Hartmut Hans-Jürgen Schmidt. (2022) de novo hATTR Amyloidose nach Domino-Lebertransplantation: Ein Fallbeispiel für den Einsatz von Patisiran. Zeitschrift für Gastroenterologie 60:11, pages 1659-1664.
Crossref
Crossref
Jagdish Chander Mohan, Jamshed Dalal, Vijay Kumar Chopra, Calambur Narasimhan, Prafulla Kerkar, Abraham Oomman, Saumitra Ray Fcsi, Anshu Rajnish Sharma, Pankaj Dougall, Shelley Simon, Atul Verma Drm & Vivek Radhakrishnan. (2022) Suspecting and diagnosing transthyretin amyloid cardiomyopathy (ATTR-CM) in India: An Indian expert consensus. Indian Heart Journal 74:6, pages 441-449.
Crossref
Crossref
Yonglong He, Wanting Zhang, Qingqing Xiao, Lifang Fan, Dechun Huang, Wei Chen & Wei He. (2022) Liposomes and liposome-like nanoparticles: From anti-fungal infection to the COVID-19 pandemic treatment. Asian Journal of Pharmaceutical Sciences 17:6, pages 817-837.
Crossref
Crossref
A. Hänselmann, D. Berliner, J. Bauersachs & U. Bavendiek. (2022) Cardiac amyloidosis—interdisciplinary approach to diagnosis and therapyKardiale Amyloidose – interdisziplinärer Ansatz bei Diagnose und Therapie. Herz 47:4, pages 324-331.
Crossref
Crossref
Hiroaki Matsushita, Yohei Misumi, Teruaki Masuda, Masamitsu Okada, Fumika Inoue, Mitsuharu Ueda & Yukio Ando. (2022) Urinary Transthyretin as a Biomarker in ATTRv Val50Met Amyloidosis. Pathophysiology 29:3, pages 333-343.
Crossref
Crossref
Hartmut H. Schmidt, Jonas Wixner, Violaine Planté‐Bordeneuve, Francisco Muñoz‐Beamud, Laura Lladó, Julian D. Gillmore, Anna Mazzeo, Xingyu Li, Seth Arum, Patrick Y. Jay, David Adams, Christel Langestroer, Anna Huesing‐Kabar, Matthias Schilling, Iyad Kabar, Rolf Backlund, Intissar Anan, Erik Nordh, Erika Uneus, Björn Pilebro, Ulrika Englund, Teresa Coelho, Marta Novais, Javier Perez, Ana Martins da Silva, Helena Pesseguerio Miranda, Joana Ramalho, Raquel Monte, Cristina Alves, Ines Cardaso, Nádia Guimaraes, Luca Gentile, Massimo Russo, Gianluca Di Bella, Amina Gaouar, Cécile Cauquil‐Michon, Ilias Kounis, Andoni Echaniz‐Laguna, Maëva Stéphant, Fetra Rakotondratafika, Yasmine Boubrit, Celine Labeyrie, Cecile Focsenaunu, Phillippe Le Corvoisier, Samar S. Ayache, Thierry Gendre, Laetitia Vervoitte, Raphaele Arrouasse, Alvaro Gragera Martinez, Cristina Borrachero, Ana Manovel, Eusebio Diaz Rodriguez, Marta Gutiérrez Gándara, Elena Fabra Jiménez, Patricia Valentina Vélez Santamaría, Yurema Martínez Vilar, Alba Cachero, Lisa Rannigan, Marianna Fontana, Richard Orrell, Sarah Louth, Liza Chacko, Sindhu Varughese, Douglas Throburn, Oliver Cohen, Steven Law, Angelique Smit & Svetla Strehina. (2022) Patisiran treatment in patients with hereditary transthyretin‐mediated amyloidosis with polyneuropathy after liver transplantation. American Journal of Transplantation 22:6, pages 1646-1657.
Crossref
Crossref
Federico Perfetto, Mattia Zampieri, Carlo Fumagalli, Marco Allinovi & Francesco Cappelli. (2022) Circulating biomarkers in diagnosis and management of cardiac amyloidosis: a review for internist. Internal and Emergency Medicine 17:4, pages 957-969.
Crossref
Crossref
Rola Khedraki. (2022) Cardiac amyloidosis: an update on diagnosis, current therapy, and future directions. Current Opinion in Cardiology 37:3, pages 272-284.
Crossref
Crossref
Xin He, Tailai Du, Tianxin Long, Xinxue Liao, Yugang Dong & Zhan-Peng Huang. (2022) Signaling cascades in the failing heart and emerging therapeutic strategies. Signal Transduction and Targeted Therapy 7:1.
Crossref
Crossref
Vanessa Cristina Cunha Sequeira, Maria Alice Penetra, Lisa Duarte, Fernanda Reis de Azevedo, Raphael Santa Rosa Sayegh, Roberto Coury Pedrosa & Márcia Waddington Cruz. (2022) Hereditary transthyretin-mediated amyloidosis with polyneuropathy: baseline anthropometric, demographic and disease characteristics of patients from a reference center. Arquivos de Neuro-Psiquiatria 80:3, pages 262-269.
Crossref
Crossref
Enrico Ammirati & Omar F AbouEzzeddine. (2022) Transthyretin amyloidosis in Western Europe: a snapshot from the THAOS registry and a call for further perspectives. European Heart Journal 43:5, pages 401-404.
Crossref
Crossref
Ming-Jen Lee, Inyoul Lee & Kai Wang. (2022) Recent Advances in RNA Therapy and Its Carriers to Treat the Single-Gene Neurological Disorders. Biomedicines 10:1, pages 158.
Crossref
Crossref
Alireza Shahryari, Zahra Nazari, Marie Saghaeian Jazi, Fatemeh Hashemi-Shahraki, Katharina Wißmiller, Weiwei Xu, Ingo Burtscher & Heiko Lickert. 2022. Comprehensive Pharmacology. Comprehensive Pharmacology
326
368
.
Aaron Yarlas, Andrew Lovley, Duncan Brown, Mark Kosinski & Montserrat Vera-Llonch. (2021) Responder analysis for neuropathic impairment and quality-of-life assessment in patients with hereditary transthyretin amyloidosis with polyneuropathy in the NEURO-TTR study. Journal of Neurology 269:1, pages 323-335.
Crossref
Crossref
Clint Asher, Andrew Guilder, Gherardo Finocchiaro, Gerry Carr‐White & Yael Rodríguez‐Guadarrama. (2022) Healthcare resource use associated with the diagnosis of transthyretin amyloidosis cardiomyopathy. Health Science Reports 5:1.
Crossref
Crossref
Svetlana N. Morozkina, Petr P. Snetkov, Roman O. Olekhnovich & Mayya V. Uspenskaya. (2021) Modern Approaches To Cardiovascular Amyloidosis Treatment. Russian Open Medical Journal 10:4.
Crossref
Crossref
Fabio Fernandes, Caio Cafezeiro, Renata Margarida do Val, Alexandra Patrícia Zilli Vieira, Wilson Marques, Edileide Barros Correia, Alzira Alves Siqueira Carvalho, Antonio Carlos Palandrini Chagas, Acary Souza Bulle Oliveira, Paulo Victor Sgobi de Souza, Wladimir Bocca Vieira de Resende Pinto, Ariane Vieira Scarlatelli Macedo, Murillo Oliveira Antunes, Pedro Vellosa Schwartzmann, Sandrigo Mangini & Marcus Vinicius Simões. (2021) Registry of Transthyretin Amyloidosis in the State of São Paulo (REACT-SP). ABC: Heart Failure & Cardiomyopathy 1:2, pages 86-89.
Crossref
Crossref
Andrew Lovley, Kimberly Raymond, Spencer D. Guthrie, Michael Pollock, Vaishali Sanchorawala & Michelle K. White. (2021) Patient-reported burden of hereditary transthyretin amyloidosis on functioning and well-being. Journal of Patient-Reported Outcomes 5:1.
Crossref
Crossref
Mohamed Abouelhoda, Dania Mohty, Islam Alayary, Brian F. Meyer, Stefan T. Arold, Bahaa M. Fadel & Dorota Monies. (2021) Established and candidate transthyretin amyloidosis variants identified in the Saudi population by data mining. Human Genomics 15:1.
Crossref
Crossref
David Adams, Vincent Algalarrondo, Michael Polydefkis, Nitasha Sarswat, Michel S. Slama & Jose Nativi-Nicolau. (2021) Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression. Orphanet Journal of Rare Diseases 16:1.
Crossref
Crossref
Montserrat Vera-Llonch, Sheila R. Reddy, Eunice Chang, Marian H. Tarbox & Michael Pollock. (2021) The patient journey toward a diagnosis of hereditary transthyretin (ATTRv) amyloidosis. Orphanet Journal of Rare Diseases 16:1.
Crossref
Crossref
Takeshi Chujo & Kazuhito Tomizawa. (2021) Human transfer RNA modopathies: diseases caused by aberrations in transfer RNA modifications. The FEBS Journal 288:24, pages 7096-7122.
Crossref
Crossref
Aaron Yarlas, Andrew Lovley, Kristen McCausland, Duncan Brown, Montserrat Vera-Llonch, Isabel Conceição, Chafic Karam, Sami Khella, Laura Obici & Márcia Waddington-Cruz. (2021) Early Data on Long-term Impact of Inotersen on Quality-of-Life in Patients with Hereditary Transthyretin Amyloidosis Polyneuropathy: Open-Label Extension of NEURO-TTR. Neurology and Therapy 10:2, pages 865-886.
Crossref
Crossref
Aldostefano Porcari, Rossana Bussani, Marco Merlo, Guerino Giuseppe Varrà, Linda Pagura, Davide Rozze & Gianfranco Sinagra. (2021) Incidence and Characterization of Concealed Cardiac Amyloidosis Among Unselected Elderly Patients Undergoing Post-mortem Examination. Frontiers in Cardiovascular Medicine 8.
Crossref
Crossref
Csilla Andrea Eötvös, Giorgia Pastiu, Iulia Zehan, Cerasela Goidescu, Roxana Chiorescu, Roxana Lazar, Florina Frîngu, Raluca Tomoaia, Monica Pop, Adrian Molnar, Sorin Pop & Dan Blendea. 2021. Cardiomyopathy - Disease of the Heart Muscle. Cardiomyopathy - Disease of the Heart Muscle.
Manish Pandey, Divya Ojha, Sakshi Bansal, Ambadas B. Rode & Geetanjali Chawla. (2021) From bench side to clinic: Potential and challenges of RNA vaccines and therapeutics in infectious diseases. Molecular Aspects of Medicine 81, pages 101003.
Crossref
Crossref
Matthew Capustin & William H. Frishman. (2021) Transthyretin Cardiac Amyloidosis and Novel Therapies to Treat This Not-so-rare Cause of Cardiomyopathy. Cardiology in Review 29:5, pages 263-273.
Crossref
Crossref
Yessar Hussain. (2021) Variable Presentation of Hereditary Transthyretin-Mediated Amyloidosis at a Single Center. Journal of Clinical Neuromuscular Disease 23:1, pages 7-17.
Crossref
Crossref
Angela Russell, Christopher Hahn, Sameer Chhibber, Lawrence Korngut & Nowell M. Fine. (2020) Utility of Neuropathy Screening for Wild-Type Transthyretin Amyloidosis Patients. Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques 48:5, pages 607-615.
Crossref
Crossref
Julian D. GillmoreEd GaneJorg TaubelJustin KaoMarianna FontanaMichael L. MaitlandJessica SeitzerDaniel O’ConnellKathryn R. WalshKristy WoodJonathan Phillips, Yuanxin XuAdam AmaralAdam P. BoydJeffrey E. CehelskyMark D. McKeeAndrew SchiermeierOlivier HarariAndrew MurphyChristos A. KyratsousBrian ZambrowiczRandy SoltysDavid E. GutsteinJohn LeonardLaura Sepp-LorenzinoDavid Lebwohl. (2021) CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. New England Journal of Medicine 385:6, pages 493-502.
Crossref
Crossref
Paolo Severino, Andrea D’Amato, Silvia Prosperi, Francesca Fanisio, Lucia Ilaria Birtolo, Bettina Costi, Lucrezia Netti, Cristina Chimenti, Carlo Lavalle, Viviana Maestrini, Massimo Mancone & Francesco Fedele. (2021) Myocardial Tissue Characterization in Heart Failure with Preserved Ejection Fraction: From Histopathology and Cardiac Magnetic Resonance Findings to Therapeutic Targets. International Journal of Molecular Sciences 22:14, pages 7650.
Crossref
Crossref
Patrick Y. Jay, Martin A. Maier, Laura Saltonstall, Lisa Duarte, Ilia Antonino & John Vest. (2021) Gene Silencing Therapeutics in Cardiology: A Review Article. International Journal of Cardiovascular Sciences.
Crossref
Crossref
M. May Zhang, Raman Bahal, Theodore P. Rasmussen, José E. Manautou & Xiao-bo Zhong. (2021) The growth of siRNA-based therapeutics: Updated clinical studies. Biochemical Pharmacology 189, pages 114432.
Crossref
Crossref
David Adams, Yukio Ando, João Melo Beirão, Teresa Coelho, Morie A. Gertz, Julian D. Gillmore, Philip N. Hawkins, Isabelle Lousada, Ole B. Suhr & Giampaolo Merlini. (2020) Expert consensus recommendations to improve diagnosis of ATTR amyloidosis with polyneuropathy. Journal of Neurology 268:6, pages 2109-2122.
Crossref
Crossref
Pablo Garcia‐Pavia, Frank Bengel, Dulce Brito, Thibaud Damy, Franz Duca, Sharmila Dorbala, Jose Nativi‐Nicolau, Laura Obici, Claudio Rapezzi, Yoshiki Sekijima & Perry M. Elliott. (2021) Expert consensus on the monitoring of transthyretin amyloid cardiomyopathy. European Journal of Heart Failure 23:6, pages 895-905.
Crossref
Crossref
Sergio Iannazzo. (2021) A comparative analysis of international health technology assessments for novel gene silencing therapies: patisiran and inotersen. Global & Regional Health Technology Assessment 8, pages 14-21.
Crossref
Crossref
Bahru A. Habtemariam, Verena Karsten, Husain Attarwala, Varun Goel, Megan Melch, Valerie A. Clausen, Pushkal Garg, Akshay K. Vaishnaw, Marianne T. Sweetser, Gabriel J. Robbie & John Vest. (2020) Single‐Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N‐acetylgalactosamine–Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects. Clinical Pharmacology & Therapeutics 109:2, pages 372-382.
Crossref
Crossref
Simina TicauGautham V. SridharanShira TsourWilliam L. CantleyAmy ChanJason A. GilbertDavid ErbeEmre AldincMary M. ReillyDavid AdamsMichael PolydefkisKevin FitzgeraldAkshay VaishnawPaul Nioi. (2021) Neurofilament Light Chain as a Biomarker of Hereditary Transthyretin-Mediated Amyloidosis. Neurology 96:3.
Crossref
Crossref
Emily R. Soper, Sabrina A. Suckiel, Giovanna T. Braganza, Amy R. Kontorovich, Eimear E. Kenny & Noura S. Abul-Husn. (2021) Genomic Screening Identifies Individuals at High Risk for Hereditary Transthyretin Amyloidosis. Journal of Personalized Medicine 11:1, pages 49.
Crossref
Crossref
Teresa Coelho, David Adams, Isabel Conceição, Márcia Waddington-Cruz, Hartmut H. Schmidt, Juan Buades, Josep Campistol, John L. Berk, Michael Polydefkis, Jing Jing Wang, Jihong Chen, Marianne T. Sweetser, Jared Gollob & Ole B. Suhr. (2020) A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis. Orphanet Journal of Rare Diseases 15:1.
Crossref
Crossref
Morie Gertz, David Adams, Yukio Ando, João Melo Beirão, Sabahat Bokhari, Teresa Coelho, Raymond L. Comenzo, Thibaud Damy, Sharmila Dorbala, Brian M. Drachman, Marianna Fontana, Julian D. Gillmore, Martha Grogan, Philip N. Hawkins, Isabelle Lousada, Arnt V. Kristen, Frederick L. Ruberg, Ole B. Suhr, Mathew S. Maurer, Jose Nativi-Nicolau, Candida Cristina Quarta, Claudio Rapezzi, Ronald Witteles & Giampaolo Merlini. (2020) Avoiding misdiagnosis: expert consensus recommendations for the suspicion and diagnosis of transthyretin amyloidosis for the general practitioner. BMC Family Practice 21:1.
Crossref
Crossref
Ivan Urits, Daniel Swanson, Michael C. Swett, Anjana Patel, Kevin Berardino, Ariunzaya Amgalan, Amnon A. Berger, Hisham Kassem, Alan D. Kaye & Omar Viswanath. (2020) A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis. Neurology and Therapy 9:2, pages 301-315.
Crossref
Crossref
Sheila R. Reddy, Eunice Chang, Marian H. Tarbox, Michael S. Broder, Ryan S. Tieu, Spencer Guthrie, Montserrat Vera-Llonch & Michael R. Pollock. (2020) The Clinical and Economic Burden of Newly Diagnosed Hereditary Transthyretin (ATTRv) Amyloidosis: A Retrospective Analysis of Claims Data. Neurology and Therapy 9:2, pages 473-482.
Crossref
Crossref
Tomizawa Kazuhito & Fan-Yan Wei. (2020) Posttranscriptional modifications in mitochondrial tRNA and its implication in mitochondrial translation and disease. The Journal of Biochemistry 168:5, pages 435-444.
Crossref
Crossref
Gabriela Spencer-Bonilla, Kevin M. Alexander & Ronald M. Witteles. (2020) Advances in the Diagnosis and Management of Transthyretin Amyloid Cardiomyopathy. Current Treatment Options in Cardiovascular Medicine 22:11.
Crossref
Crossref
Hollis Lin, Madeline Merkel, Cecilia Hale & Jing L Marantz. (2020) Experience of patisiran with transthyretin stabilizers in patients with hereditary transthyretin-mediated amyloidosis. Neurodegenerative Disease Management 10:5, pages 289-300.
Crossref
Crossref
Jorge Penalver, Maxwell Ambrosino, Hee D. Jeon, Akanksha Agrawal, Napatt Kanjanahattakij, Marie Pitteloud, Jessica Stempel & Aman Amanullah. (2020) Transthyretin Cardiac Amyloidosis and Aortic Stenosis: Connection and Therapeutic Implications. Current Cardiology Reviews 16:3, pages 221-230.
Crossref
Crossref
Taro Yamashita, Mitsuharu Ueda, Haruki Koike, Yoshiki Sekijima, Tsuneaki Yoshinaga, Minori Kodaira, Masahisa Katsuno, Gen Sobue, Xiaoping Zhang, Matthew T. White, Marianne T. Sweetser, Jing Jing Wang & Yukio Ando. (2020) Patisiran, an RNAi therapeutic for patients with hereditary transthyretin‐mediated amyloidosis: Sub‐analysis in Japanese patients from the APOLLO study. Neurology and Clinical Neuroscience 8:5, pages 251-260.
Crossref
Crossref
Varun GoelNathalie H. GosselinClaudia JompheXiaoping ZhangJean-Francois MarierGabriel J. Robbie. (2020) Population Pharmacokinetic–Pharmacodynamic Model of Serum Transthyretin Following Patisiran Administration. Nucleic Acid Therapeutics 30:3, pages 143-152.
Crossref
Crossref
Alejandra González‐Duarte, Isabel Conceição, Leslie Amass, Marc F. Botteman, John A. Carter & Michelle Stewart. (2020) Impact of Non-Cardiac Clinicopathologic Characteristics on Survival in Transthyretin Amyloid Polyneuropathy. Neurology and Therapy 9:1, pages 135-149.
Crossref
Crossref
Daniel P. Judge, Arnt V. Kristen, Martha Grogan, Mathew S. Maurer, Rodney H. Falk, Mazen Hanna, Julian Gillmore, Pushkal Garg, Akshay K. Vaishnaw, Jamie Harrop, Christine Powell, Verena Karsten, Xiaoping Zhang, Marianne T. Sweetser, John Vest & Philip N. Hawkins. (2020) Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR). Cardiovascular Drugs and Therapy 34:3, pages 357-370.
Crossref
Crossref
Huang Zuo, Xiaohong Su, Yixiao Jin, Cheng Zhang, Lelin Wang & Lin Yang. (2020) Transthyretin Regulated by linc00657/miR-205-5p Promoted Cholesterol Metabolism by Inducing SREBP2-HMGCR and Inhibiting LXRα-CYP7A1. Archives of Medical Research 51:4, pages 317-326.
Crossref
Crossref
Xiaoping Zhang, Varun Goel & Gabriel J. Robbie. (2019) Pharmacokinetics of Patisiran, the First Approved RNA Interference Therapy in Patients With Hereditary Transthyretin‐Mediated Amyloidosis. The Journal of Clinical Pharmacology 60:5, pages 573-585.
Crossref
Crossref
Phei Er Saw & Er-Wei Song. (2019) siRNA therapeutics: a clinical reality. Science China Life Sciences 63:4, pages 485-500.
Crossref
Crossref
Teresa Coelho, Aaron Yarlas, Marcia Waddington-Cruz, Michelle K. White, Asia Sikora Kessler, Andrew Lovley, Michael Pollock, Spencer Guthrie, Elizabeth J. Ackermann, Steven G. Hughes, Chafic Karam, Sami Khella, Morie Gertz, Giampaolo Merlini, Laura Obici, Hartmut H. Schmidt, Michael Polydefkis, P. James B. Dyck, Thomas H. Brannagan III, Isabel Conceição, Merrill D. Benson & John L. Berk. (2019) Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis. Journal of Neurology 267:4, pages 1070-1079.
Crossref
Crossref
Coreen Schwartzlow & Mohamed Kazamel. (2020) Hereditary Transthyretin Amyloidosis: Clinical Presentation and Management Updates. Journal of Clinical Neuromuscular Disease 21:3, pages 144-156.
Crossref
Crossref
Iuliana Vaxman, Angela Dispenzieri, Eli Muchtar & Morie Gertz. (2020) New developments in diagnosis, risk assessment and management in systemic amyloidosis. Blood Reviews 40, pages 100636.
Crossref
Crossref
Alejandra González-Duarte, John L. Berk, Dianna Quan, Michelle L. Mauermann, Hartmut H. Schmidt, Michael Polydefkis, Márcia Waddington-Cruz, Mitsuharu Ueda, Isabel M. Conceição, Arnt V. Kristen, Teresa Coelho, Cécile A. Cauquil, Céline Tard, Madeline Merkel, Emre Aldinc, Jihong Chen, Marianne T. Sweetser, Jing Jing Wang & David Adams. (2019) Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis. Journal of Neurology 267:3, pages 703-712.
Crossref
Crossref
Simoneide S. Titze-de-Almeida, Pedro Renato de Paula Brandão, Ingrid Faber & Ricardo Titze-de-Almeida. (2019) Leading RNA Interference Therapeutics Part 1: Silencing Hereditary Transthyretin Amyloidosis, with a Focus on Patisiran. Molecular Diagnosis & Therapy 24:1, pages 49-59.
Crossref
Crossref
Andrew J. Einstein, Igor Shuryak, Adam Castaño, Akiva Mintz, Mathew S. Maurer & Sabahat Bokhari. (2018) Estimating cancer risk from 99mTc pyrophosphate imaging for transthyretin cardiac amyloidosis. Journal of Nuclear Cardiology 27:1, pages 215-224.
Crossref
Crossref
Sarah Bajan & Gyorgy Hutvagner. (2020) RNA-Based Therapeutics: From Antisense Oligonucleotides to miRNAs. Cells 9:1, pages 137.
Crossref
Crossref
Adam S. Fisch, Florian J. Fintelmann, Maria L. Garcia-Moliner, Henning A. Gaissert, Inga Lennes, James N. Butera, James R. Stone & Lida P. Hariri. (2020) Systemic Amyloidosis Mimicking Lung Cancer. American Journal of Respiratory and Critical Care Medicine 201:1, pages 107-108.
Crossref
Crossref
Foteini Theodorakakou, Despina Fotiou, Meletios A. Dimopoulos & Efstathios Kastritis. (2020) Solid Organ Transplantation in Amyloidosis. Acta Haematologica 143:4, pages 352-364.
Crossref
Crossref
Dominik Witzigmann, Jayesh A. Kulkarni, Jerry Leung, Sam Chen, Pieter R. Cullis & Roy van der Meel. (2020) Lipid nanoparticle technology for therapeutic gene regulation in the liver. Advanced Drug Delivery Reviews 159, pages 344-363.
Crossref
Crossref
Jin-Ping Li & Xiao Zhang. 2020. Heparanase. Heparanase
631
645
.
Xiaoping Zhang, Varun Goel, Husain Attarwala, Marianne T. Sweetser, Valerie A. Clausen & Gabriel J. Robbie. (2019) Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure‐Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin‐Mediated (hATTR) Amyloidosis. The Journal of Clinical Pharmacology 60:1, pages 37-49.
Crossref
Crossref
P. James B. Dyck, A. González-Duarte, L. Obici, M. Polydefkis, J.F. Wiesman, I. Antonino, W.J. Litchy & Peter J. Dyck. (2019) Development of measures of polyneuropathy impairment in hATTR amyloidosis: From NIS to mNIS + 7. Journal of the Neurological Sciences 405, pages 116424.
Crossref
Crossref
Alessandra Corazza, Guglielmo VeronaChristopher A. Waudby, P. Patrizia Mangione, Ryan BinghamIain UingsDiana CanettiPaola NocerinoGraham W. TaylorMark B. PepysJohn ChristodoulouVittorio Bellotti. (2019) Binding of Monovalent and Bivalent Ligands by Transthyretin Causes Different Short- and Long-Distance Conformational Changes. Journal of Medicinal Chemistry 62:17, pages 8274-8283.
Crossref
Crossref
Danielle Brandman, Hollis Lin, Anastasia McManus, Sonalee Agarwal, Larry M. Gache, William Irish, Jared Gollob & Saša A Živković. (2019) Evaluating Prognostic Factors for Liver Transplantation Among United States Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis Using National Registry Data. Progress in Transplantation 29:3, pages 213-219.
Crossref
Crossref
Mathew S. MaurerSabahat BokhariThibaud DamySharmila DorbalaBrian M. DrachmanMarianna FontanaMartha GroganArnt V. KristenIsabelle LousadaJose Nativi-NicolauCandida Cristina QuartaClaudio RapezziFrederick L. RubergRonald WittelesGiampaolo Merlini. (2019) Expert Consensus Recommendations for the Suspicion and Diagnosis of Transthyretin Cardiac Amyloidosis. Circulation: Heart Failure 12:9.
Crossref
Crossref
Saša A. ŽivkovićDiana Mnatsakanova & David Lacomis. (2019) Phenotypes of Late-Onset Transthyretin Amyloid Neuropathy: A Diagnostic Challenge. Journal of Clinical Neuromuscular Disease 21:1, pages 1-6.
Crossref
Crossref
Aaron Yarlas, Morie A. Gertz, Noel R. Dasgupta, Laura Obici, Michael Pollock, Elizabeth J. Ackermann, Andrew Lovley, Asia Sikora Kessler, Pankaj A. Patel, Michelle K. White & Spencer D. Guthrie. (2019) Burden of hereditary transthyretin amyloidosis on quality of life. Muscle & Nerve 60:2, pages 169-175.
Crossref
Crossref
Kathleen W. Zhang, Keith E. Stockerl-Goldstein & Daniel J. Lenihan. (2019) Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis. JACC: Basic to Translational Science 4:3, pages 438-448.
Crossref
Crossref
Yvette N. Lamb & Emma D. Deeks. (2019) Tafamidis: A Review in Transthyretin Amyloidosis with Polyneuropathy. Drugs 79:8, pages 863-874.
Crossref
Crossref
Mahima Kapoor, Alexander M. Rossor, Matilde Laura & Mary M. Reilly. (2019) Clinical Presentation, Diagnosis and Treatment of TTR Amyloidosis. Journal of Neuromuscular Diseases 6:2, pages 189-199.
Crossref
Crossref
Carmen Lahuerta Pueyo, Miguel Ángel Aibar Arregui, Anyuli Gracia Gutierrez, Esperanza Bueno Juana & Sebastián Menao Guillén. (2019) Estimating the prevalence of allelic variants in the transthyretin gene by analysing large-scale sequencing data. European Journal of Human Genetics 27:5, pages 783-791.
Crossref
Crossref
Danah Al Shaer, Othman Al Musaimi, Fernando Albericio & Beatriz de la Torre. (2019) 2018 FDA Tides Harvest. Pharmaceuticals 12:2, pages 52.
Crossref
Crossref
Ridwan Babatunde Ibrahim, Yo-Tsen Liu, Ssu-Yu Yeh & Jin-Wu Tsai. (2019) Contributions of Animal Models to the Mechanisms and Therapies of Transthyretin Amyloidosis. Frontiers in Physiology 10.
Crossref
Crossref
Alejandra Gonzalez-Duarte. (2018) Autonomic involvement in hereditary transthyretin amyloidosis (hATTR amyloidosis). Clinical Autonomic Research 29:2, pages 245-251.
Crossref
Crossref
Marta Spodzieja, Sylwia Rodziewicz-Motowidło & Aneta Szymanska. (2019) Hyphenated Mass Spectrometry Techniques in the Diagnosis of Amyloidosis. Current Medicinal Chemistry 26:1, pages 104-120.
Crossref
Crossref
Federico Perfetto, Francesco Cappelli, Silvia Farsetti, Elio Dimarcantonio, Silvia Casagrande, Massimo Di Gioia, Marco Di Girolamo & Franco Bergesio. (2019) Amiloidosi da transtiretina (ATTR): l’altra faccia della medaglia. Giornale di Tecniche Nefrologiche e Dialitiche 31:1, pages 12-21.
Crossref
Crossref
Arnt V Kristen, Senda Ajroud-Driss, Isabel Conceição, Peter Gorevic, Theodoros Kyriakides & Laura Obici. (2019) Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis. Neurodegenerative Disease Management 9:1, pages 5-23.
Crossref
Crossref
Scott D. SolomonDavid AdamsArnt KristenMartha GroganAlejandra González-DuarteMathew S. MaurerGiampaolo MerliniThibaud DamyMichel S. SlamaThomas H. BrannaganIIIIIIAngela DispenzieriJohn L. BerkAmil M. ShahPushkal GargAkshay VaishnawVerena KarstenJihong ChenJared GollobJohn VestOle Suhr. (2019) Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis. Circulation 139:4, pages 431-443.
Crossref
Crossref
Kristin MickleKaren E. LasserJeffrey S. HochLauren E. CiprianoWilliam B. DreitleinSteven D. Pearson. (2019) The Effectiveness and Value of Patisiran and Inotersen for Hereditary Transthyretin Amyloidosis. Journal of Managed Care & Specialty Pharmacy 25:1, pages 10-15.
Crossref
Crossref
Yuya Hayashi & Hirofumi Jono. (2018) Recent Advances in Oligonucleotide-Based Therapy for Transthyretin Amyloidosis: Clinical Impact and Future Prospects. Biological and Pharmaceutical Bulletin 41:12, pages 1737-1744.
Crossref
Crossref
Yoshiki Sekijima, Mitsuharu Ueda, Haruki Koike, Sonoko Misawa, Tomonori Ishii & Yukio Ando. (2018) Diagnosis and management of transthyretin familial amyloid polyneuropathy in Japan: red-flag symptom clusters and treatment algorithm. Orphanet Journal of Rare Diseases 13:1.
Crossref
Crossref
P. V. Ershov, Yu. V. Mezentsev, E. O. Yablokov, L. A. Kaluzhskiy, A. V. Florinskaya, O. V. Gnedenko, V. G. Zgoda, I. V. Vakhrushev, O. S. Raeva, K. N. Yarygin, A. A. Gilep, S. A. Usanov, A. E. Medvedev & A. S. Ivanov. (2019) Direct Molecular Fishing of Protein Partners for Proteins Encoded by Genes of Human Chromosome 18 in HepG2 Cell Lysate. Russian Journal of Bioorganic Chemistry 44:6, pages 759-768.
Crossref
Crossref
N. Andrés, J.J. Poza & J.F. Martí Massó. (2018) Familial amyloidosis with polyneuropathy type 1 caused by transthyretin mutation Val50Met (Val30Met): 4 cases in a non-endemic area. Neurología (English Edition) 33:9, pages 583-589.
Crossref
Crossref
N. Andrés, J.J. Poza & J.F. Martí Massó. (2018) Polineuropatía amiloidótica familiar I por mutación Val50Met (Val30Met) en el gen de la transtiretina. Neurología 33:9, pages 583-589.
Crossref
Crossref
Sheridan M. Hoy. (2018) Patisiran: First Global Approval. Drugs 78:15, pages 1625-1631.
Crossref
Crossref
J. Wixner, H. Törnblom, P. Karling, I. Anan & G. Lindberg. (2018) Abnormal small bowel motility in patients with hereditary transthyretin amyloidosis. Neurogastroenterology & Motility 30:9, pages e13354.
Crossref
Crossref
Susan J. Keam. (2018) Inotersen: First Global Approval. Drugs 78:13, pages 1371-1376.
Crossref
Crossref
David AdamsAlejandra Gonzalez-DuarteWilliam D. O’RiordanChih-Chao YangMitsuharu UedaArnt V. KristenIvailo TournevHartmut H. SchmidtTeresa CoelhoJohn L. BerkKon-Ping LinGiuseppe VitaShahram AttarianViolaine Planté-BordeneuveMichelle M. MezeiJosep M. CampistolJuan BuadesThomas H. BrannaganIIIIIIByoung J. KimJeeyoung OhYesim ParmanYoshiki SekijimaPhilip N. HawkinsScott D. SolomonMichael PolydefkisPeter J. DyckPritesh J. GandhiSunita GoyalJihong ChenAndrew L. StrahsSaraswathy V. NochurMarianne T. SweetserPushkal P. GargAkshay K. VaishnawJared A. GollobOle B. Suhr. (2018) Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. New England Journal of Medicine 379:1, pages 11-21.
Crossref
Crossref
Scot Walker. (2018) New Medications in the Treatment of Hereditary Transthyretin Amyloidosis. Hospital Pharmacy 53:4, pages 236-238.
Crossref
Crossref
Aaron D. SpringerSteven F. Dowdy. (2018) GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics. Nucleic Acid Therapeutics 28:3, pages 109-118.
Crossref
Crossref
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.
Crossref
Crossref
Cherie N. Dahm, R. Frank Cornell & Daniel J. Lenihan. (2018) Advances in Treatment of Cardiac Amyloid. Current Treatment Options in Cardiovascular Medicine 20:5.
Crossref
Crossref
Hartmut H. Schmidt, Márcia Waddington-Cruz, Marc F. Botteman, John A. Carter, Avijeet S. Chopra, Markay Hopps, Michelle Stewart, Shari Fallet & Leslie Amass. (2018) Estimating the global prevalence of transthyretin familial amyloid polyneuropathy. Muscle & Nerve 57:5, pages 829-837.
Crossref
Crossref
Hisae Sumi-Akamaru, Masaki Eto, Amane Yamauchi, Takuya Uehara, Keita Kakuda, Konen Obayashi, Shinsuke Kato, Takashi Naka & Hideki Mochizuki. (2018) Evidence that glial cells attenuate G47R transthyretin accumulation in the central nervous system. Neuropathology 38:1, pages 11-21.
Crossref
Crossref
Nazila Rad & Said R Beydoun. (2018) Late-onset Hereditary Transthyretin Amyloidosis in Two Patients with Acquired Demyelinating Features. US Neurology 14:2, pages 98.
Crossref
Crossref
Yuriko Wakita, Takashi Sato, Keisuke Chosa, Mary Ann Suico, Ryoko Sasaki, Shingo Kawano, Nami Hashimoto, Yuriko Teranishi, Yoshiki Imai, Hiroshi Morioka, Tsuyoshi Shuto & Hirofumi Kai. (2018) Characterization of Non-amyloidogenic G101S Transthyretin. Biological and Pharmaceutical Bulletin 41:4, pages 628-636.
Crossref
Crossref
Todd D Levine. (2018) Small Fiber Neuropathy: Disease Classification Beyond Pain and Burning. Journal of Central Nervous System Disease 10, pages 117957351877170.
Crossref
Crossref
Omar K. Siddiqi & Frederick L. Ruberg. (2018) Cardiac amyloidosis: An update on pathophysiology, diagnosis, and treatment. Trends in Cardiovascular Medicine 28:1, pages 10-21.
Crossref
Crossref
Feng Zhu & Kai Huang. 2018. Heart Genomics. Heart Genomics
337
374
.
Claudio Rapezzi, Christian Gagliardi, Fabrizio Salvi, Ilaria Bartolomei, Candida Cristina Quarta & Agnese Milandri. 2018. Cardiovascular Genetics and Genomics. Cardiovascular Genetics and Genomics
545
577
.
João L. Cavalcante, Shasank Rijal, Islam Abdelkarim, Andrew D. Althouse, Michael S. Sharbaugh, Yaron Fridman, Prem Soman, Daniel E. Forman, John T. Schindler, Thomas G. Gleason, Joon S. Lee & Erik B. Schelbert. (2017) Cardiac amyloidosis is prevalent in older patients with aortic stenosis and carries worse prognosis. Journal of Cardiovascular Magnetic Resonance 19:1.
Crossref
Crossref
David Adams, Ole B. Suhr, Peter J. Dyck, William J. Litchy, Raina G. Leahy, Jihong Chen, Jared Gollob & Teresa Coelho. (2017) Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy. BMC Neurology 17:1.
Crossref
Crossref
Taryn Youngstein, Janet A. Gilbertson, David F. Hutt, Mark R. E. Coyne, Tamer Rezk, Richa Manwani, Candida C. Quarta, Helen J. Lachmann, Julian D. Gillmore, Huw Beynon, Nicholas Goddard & Philip N. Hawkins. (2017) Carpal Tunnel Biopsy Identifying Transthyretin Amyloidosis. Arthritis & Rheumatology 69:10, pages 2051-2051.
Crossref
Crossref
Ricardo Titze-de-Almeida, Catherine David & Simoneide Souza Titze-de-Almeida. (2017) The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market. Pharmaceutical Research 34:7, pages 1339-1363.
Crossref
Crossref
Adam Castaño, Daniel K. Manson, Mathew S. Maurer & Sabahat Bokhari. (2017) Transthyretin Cardiac Amyloidosis in Older Adults: Optimizing Cardiac Imaging to the Corresponding Diagnostic and Management Goal. Current Cardiovascular Risk Reports 11:6.
Crossref
Crossref
Mathew S. MaurerPerry ElliottRaymond ComenzoMarc SemigranClaudio Rapezzi. (2017) Addressing Common Questions Encountered in the Diagnosis and Management of Cardiac Amyloidosis. Circulation 135:14, pages 1357-1377.
Crossref
Crossref
Natalie J. Galant, Per Westermark, Jeffrey N. HigakiAvijit Chakrabartty. (2017) Transthyretin amyloidosis: an under-recognized neuropathy and cardiomyopathy. Clinical Science 131:5, pages 395-409.
Crossref
Crossref
Yuanyu Huang. (2017) Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics. Molecular Therapy - Nucleic Acids 6, pages 116-132.
Crossref
Crossref
Kate McKeage, Katherine A. Lyseng-Williamson & Lesley J. Scott. (2016) Tafamidis in transthyretin amyloidosis: a guide to its use in delaying peripheral neurological impairment in patients with stage 1 polyneuropathy. Drugs & Therapy Perspectives 33:2, pages 47-53.
Crossref
Crossref
Anastasia Khvorova. (2017) Oligonucleotide Therapeutics — A New Class of Cholesterol-Lowering Drugs. New England Journal of Medicine 376:1, pages 4-7.
Crossref
Crossref
Danielle L. Brunjes, Adam Castano, Autumn Clemons, Jonah Rubin & Mathew S. Maurer. (2016) Transthyretin Cardiac Amyloidosis in Older Americans. Journal of Cardiac Failure 22:12, pages 996-1003.
Crossref
Crossref
Srijita Sen-Chowdhry, Daniel Jacoby, James C. Moon & William J. McKenna. (2016) Update on hypertrophic cardiomyopathy and a guide to the guidelines. Nature Reviews Cardiology 13:11, pages 651-675.
Crossref
Crossref
Genki Suenaga, Tokunori Ikeda, Yoshihiro Komohara, Koutaro Takamatsu, Tatsuyuki Kakuma, Masayoshi Tasaki, Yohei Misumi, Mitsuharu Ueda, Takaaki Ito, Satoru Senju & Yukio Ando. (2016) Involvement of Macrophages in the Pathogenesis of Familial Amyloid Polyneuropathy and Efficacy of Human iPS Cell-Derived Macrophages in Its Treatment. PLOS ONE 11:10, pages e0163944.
Crossref
Crossref
Irena Roterman, Mateusz Banach, Barbara Kalinowska & Leszek Konieczny. (2016) Influence of the Aqueous Environment on Protein Structure—A Plausible Hypothesis Concerning the Mechanism of Amyloidogenesis. Entropy 18:10, pages 351.
Crossref
Crossref
Kevin M. AlexanderRodney H. Falk. (2016) V122I TTR Cardiac Amyloidosis in Patients of African Descent. Circulation: Heart Failure 9:9.
Crossref
Crossref