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Drug Design, Development and Therapy Volume 15, 2021 - Issue
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Review

Drug Discovery and Development in Rare Diseases: Taking a Closer Look at the Tafamidis Story

Arianna Burton1 Medical Affairs, Pfizer Inc, New York, NY, USA
,
Adam Castaño1 Medical Affairs, Pfizer Inc, New York, NY, USACorrespondence[email protected]
,
Marianna Bruno1 Medical Affairs, Pfizer Inc, New York, NY, USA
,
Steve Riley2 Clinical Pharmacology, Pfizer Inc, Groton, CT, USA
,
Jennifer Schumacher1 Medical Affairs, Pfizer Inc, New York, NY, USA
,
Marla B Sultan3 Global Product Development, Pfizer Inc, New York, NY, USA
,
Sandi See Tai4 Global Product Development, Pfizer Inc, Collegeville, PA, USA
,
Daniel P Judge5 Department of Medicine, Medical University of South Carolina, Charleston, SC, USA
,
Jignesh K Patel6 Department of Cardiology, Cedars-Sinai Smidt Heart Institute, Los Angeles, CA, USA
&
Jeffery W Kelly7 Department of Chemistry, The Scripps Research Institute, La Jolla, CA, USA
 show all
Pages 1225-1243 | Published online: 18 Mar 2021

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Catarina Falcão de Campos & Isabel Conceição. (2023) Updated Evaluation of the Safety, Efficacy and Tolerability of Tafamidis in the Treatment of Hereditary Transthyretin Amyloid Polyneuropathy. Drug, Healthcare and Patient Safety 15, pages 51-62.
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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.
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Articles from other publishers (20)

Jeffrey S. Barrett, Alexandre Betourne, Ramona L. Walls, Kara Lasater, Scott Russell, Amanda Borens, Shlok Rohatagi & Will Roddy. (2023) The future of rare disease drug development: the rare disease cures accelerator data analytics platform (RDCA-DAP). Journal of Pharmacokinetics and Pharmacodynamics 50:6, pages 507-519.
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M. Smerikarova, S. Bozhanov, I. Tournev & V. Maslarska. (2023) Variation of tafamidis plasma levels during the treatment of TTR amyloidosis patients with Glu89Gln mutation. European Journal of Clinical Pharmacology 79:12, pages 1657-1664.
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Robert J. Pignolo, Mona Al Mukaddam, Geneviève Baujat, Matthew A. Brown, Carmen De Cunto, Edward C. Hsiao, Richard Keen, Kim-Hanh Le Quan Sang, Donna R. Grogan, Rose Marino, Andrew R. Strahs & Frederick S. Kaplan. (2023) Study methodology and insights from the palovarotene clinical development program in fibrodysplasia ossificans progressiva. BMC Medical Research Methodology 23:1.
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Anson T.C. Lau, Robert J. DiDomenico & Kibum Kim. (2023) Cost-effectiveness of systematic screening and treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) in patients with heart failure with preserved ejection fraction (HFpEF) in United States. International Journal of Cardiology, pages 131598.
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Nghia Nguyen Thi Minh, Afshan Begum, Jun Zhang, Petter Leira, Yogesh Todarwal, Mathieu Linares, Patrick Norman, Dean Derbyshire, Eleonore von Castelmur, Mikael Lindgren, Per Hammarström & Carolin König. (2023) Binding of a Pyrene-Based Fluorescent Amyloid Ligand to Transthyretin: A Combined Crystallographic and Molecular Dynamics Study. The Journal of Physical Chemistry B 127:30, pages 6628-6635.
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Carmine Pecoraro, Tiziana Fioretti, Assunta Perruno, Antonella Klain, Daniela Cioffi, Adelaide Ambrosio, Diego Passaro, Luigi Annicchiarico Petruzzelli, Carmela Di Domenico, Domenico de Girolamo, Sabrina Vallone, Fabio Cattaneo, Rosario Ammendola & Gabriella Esposito. (2023) De Novo Large Deletions in the PHEX Gene Caused X-Linked Hypophosphataemic Rickets in Two Italian Female Infants Successfully Treated with Burosumab. Diagnostics 13:15, pages 2552.
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Mineyuki Mizuguchi, Takeshi Yokoyama, Takuya Okada, Yusuke Nakagawa, Kanako Fujii, Yuko Nabeshima & Naoki Toyooka. (2023) Benziodarone and 6-hydroxybenziodarone are potent and selective inhibitors of transthyretin amyloidogenesis. Bioorganic & Medicinal Chemistry 90, pages 117370.
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Ai Peng, Xue Fan, Linling Zou, Huan Chen & Jin Xiang. (2023) Trend of clinical trials of new drugs for rare diseases in China in recent 10 years. Orphanet Journal of Rare Diseases 18:1.
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Jeffery Budweg, Mohammad Al-Ani, Yazan Assaf, Alex Parker & Juan Aranda. (2023) Amyloid transthyretin cardiac amyloidosis with different manifestations, test findings and types. BMJ Case Reports 16:4, pages e250972.
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Afshan Begum, Jun Zhang, Dean Derbyshire, Xiongyu Wu, Peter Konradsson, Per Hammarström & Eleonore von Castelmur. (2023) Transthyretin Binding Mode Dichotomy of Fluorescent trans -Stilbene Ligands . ACS Chemical Neuroscience 14:5, pages 820-828.
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Yanhong Zhou, Yujie Zhao, Greg Cicconetti, Yunming Mu, Ying Yuan, Li Wang, Sudhir Penugonda & Zeena Salman. (2022) AIDE : Adaptive intrapatient dose escalation designs to accelerate Phase I clinical trials . Pharmaceutical Statistics 22:2, pages 300-311.
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Ming-Yue XU, Wei HUANG, Kan ZHANG, Dai ZHANG & Yun-Li XING. (2023) A misdiagnosed transthyretin in an elderly with myocardial hypertrophy and atrioventricular block: a case report. Journal of Geriatric Cardiology 20:2, pages 150-154.
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Mineyuki Mizuguchi, Yusuke Nakagawa, Kishin Inui, Wakana Katayama, Yurika Sawai, Ayaka Shimane, Ryota Kitakami, Takuya Okada, Yuko Nabeshima, Takeshi Yokoyama, Kayoko Kanamitsu, Shinsaku Nakagawa & Naoki Toyooka. (2022) Chlorinated Naringenin Analogues as Potential Inhibitors of Transthyretin Amyloidogenesis. Journal of Medicinal Chemistry 65:24, pages 16218-16233.
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Michael Pallin & Jane Willis. (2022) Innovative cystic fibrosis drug development: A perspective. Respirology 27:12, pages 1015-1017.
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Mazen Hanna, Nowell M. Fine, Balarama Gundapaneni, Marla B. Sultan & Ronald M. Witteles. (2022) Improvements in Efficacy Measures With Tafamidis in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial. JACC: Advances 1:5, pages 100148.
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Maria M. Picken. (2022) Current Understanding of Systemic Amyloidosis and Underlying Disease Mechanisms. The American Journal of Cardiology 185, pages S2-S10.
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Jing Liu, Jeffrey S. Barrett, Efthimia T. Leonardi, Lucy Lee, Satrajit Roychoudhury, Yong Chen & Panayiota Trifillis. (2022) Natural History and Real‐World Data in Rare Diseases: Applications, Limitations, and Future Perspectives. The Journal of Clinical Pharmacology 62:S2.
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Thomas Löhr, Kai Kohlhoff, Gabriella T. Heller, Carlo Camilloni & Michele Vendruscolo. (2022) A Small Molecule Stabilizes the Disordered Native State of the Alzheimer’s Aβ Peptide. ACS Chemical Neuroscience 13:12, pages 1738-1745.
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Jaleh Mesgarzadeh, Isabelle Romine, Ethan Smith-Cohen, Julia Grandjean, Jeffery Kelly, Joseph Genereux & R. Wiseman. (2022) ATF6 Activation Reduces Amyloidogenic Transthyretin Secretion through Increased Interactions with Endoplasmic Reticulum Proteostasis Factors. Cells 11:10, pages 1661.
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Jin-Beom Si, Bokyung Kim & Jin Hae Kim. (2021) Transthyretin Misfolding, A Fatal Structural Pathogenesis Mechanism. International Journal of Molecular Sciences 22:9, pages 4429.
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