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Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration Volume 20, 2019 - Issue 7-8
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Clinical Trials

A phase III trial of tirasemtiv as a potential treatment for amyotrophic lateral sclerosis

Jeremy M. ShefnerDepartment of Neurology, Barrow Neurological Institute, Phoenix, AZ, USA, ;Department of Neurology, University of Arizona, Phoenix, AZ, USA, Correspondence[email protected]
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Merit E. CudkowiczDepartment of Neurology, Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, MA, USA, View further author information
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Orla HardimanAcademic Unit of Neurology, Trinity Biomedical Sciences Institute, Trinity College, Dublin, Ireland, View further author information
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Bettina M. CockroftCytokinetics, Inc, South San Francisco, CA, USA, and View further author information
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Jacqueline H. LeeCytokinetics, Inc, South San Francisco, CA, USA, and View further author information
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Fady I. MalikCytokinetics, Inc, South San Francisco, CA, USA, and View further author information
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Lisa MengCytokinetics, Inc, South San Francisco, CA, USA, and View further author information
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Stacy A. RudnickiCytokinetics, Inc, South San Francisco, CA, USA, and View further author information
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Andrew A. WolffCytokinetics, Inc, South San Francisco, CA, USA, and View further author information
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Jinsy A. AndrewsThe Neurological Institute, Columbia University, New York, NY, USAView further author information
& on behalf of the VITALITY-ALS STUDY GROUP show all
Pages 584-594 | Received 08 Feb 2019, Accepted 22 Apr 2019, Published online: 13 May 2019

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. (2023) Theme 10 - Disease Stratification and Phenotyping of Patients. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 24:sup1, pages 230-244.
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Danielle Beaulieu, James D. Berry, Sabrina Paganoni, Jonathan D. Glass, Christina Fournier, Jonavelle Cuerdo, Mark Schactman & David L. Ennist. (2021) Development and validation of a machine-learning ALS survival model lacking vital capacity (VC-Free) for use in clinical trials during the COVID-19 pandemic. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 22:sup1, pages 22-32.
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Articles from other publishers (22)

Nicholas J. Maragakis, Mamede de Carvalho & Michael D. Weiss. (2023) Therapeutic targeting of ALS pathways: Refocusing an incomplete picture . Annals of Clinical and Translational Neurology 10:11, pages 1948-1971.
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Abdul Rashid Issahaku, Mahmoud A. A. Ibrahim, Namutula Mukelabai & Mahmoud E. S. Soliman. (2023) Intermolecular And Dynamic Investigation of The Mechanism of Action of Reldesemtiv on Fast Skeletal Muscle Troponin Complex Toward the Treatment of Impaired Muscle Function. The Protein Journal 42:4, pages 263-275.
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Wout J. Claassen, Rianne J. Baelde, Ricardo A. Galli, Josine M. de Winter & Coen A. C. Ottenheijm. (2023) Small molecule drugs to improve sarcomere function in those with acquired and inherited myopathies. American Journal of Physiology-Cell Physiology 325:1, pages C60-C68.
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Jeremy M. Shefner, Richard Bedlack, Jinsy A. Andrews, James D. Berry, Robert Bowser, Robert Brown, Jonathan D. Glass, Nicholas J. Maragakis, Timothy M. Miller, Jeffrey D. Rothstein & Merit E. Cudkowicz. (2022) Amyotrophic Lateral Sclerosis Clinical Trials and Interpretation of Functional End Points and Fluid Biomarkers. JAMA Neurology 79:12, pages 1312.
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Lee J. Martin, Danya A. Adams, Mark V. Niedzwiecki & Margaret Wong. (2022) Aberrant DNA and RNA Methylation Occur in Spinal Cord and Skeletal Muscle of Human SOD1 Mouse Models of ALS and in Human ALS: Targeting DNA Methylation Is Therapeutic. Cells 11:21, pages 3448.
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Mohammed Khamaysa & Pierre-François Pradat. (2022) Status of ALS Treatment, Insights into Therapeutic Challenges and Dilemmas. Journal of Personalized Medicine 12:10, pages 1601.
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Cristina Morelli, Gessica Ingrasciotta, Daniel Jacoby, Ahmad Masri & Iacopo Olivotto. (2022) Sarcomere protein modulation: The new frontier in cardiovascular medicine and beyond. European Journal of Internal Medicine 102, pages 1-7.
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Zabed Mahmud, Svetlana Tikunova, Natalya Belevych, Cory S. Wagg, Pavel Zhabyeyev, Philip B. Liu, David V. Rasicci, Christopher M. Yengo, Gavin Y. Oudit, Gary D. Lopaschuk, Peter J. Reiser, Jonathan P. Davis & Peter M. Hwang. (2022) Small Molecule RPI-194 Stabilizes Activated Troponin to Increase the Calcium Sensitivity of Striated Muscle Contraction. Frontiers in Physiology 13.
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Scott E. Collibee, Gustave Bergnes, Chihyuan Chuang, Luke Ashcraft, Jeffrey Gardina, Marc Garard, Chris R. Jamison, Kevin Lu, Pu-Ping Lu, Alexander Muci, Antonio Romero, Ellen Valkevich, Wenyue Wang, Jeffrey Warrington, Bing Yao, Nickie Durham, James Hartman, Anna Marquez, Aaron Hinken, Julia Schaletzky, Donghong Xu, Darren T. Hwee, David Morgans, Fady I. Malik & Bradley P. Morgan. (2021) Discovery of Reldesemtiv, a Fast Skeletal Muscle Troponin Activator for the Treatment of Impaired Muscle Function. Journal of Medicinal Chemistry 64:20, pages 14930-14941.
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Martijn van de Locht, Tamara C. Borsboom, Josine M. Winter & Coen A. C. Ottenheijm. (2021) Troponin Variants in Congenital Myopathies: How They Affect Skeletal Muscle Mechanics. International Journal of Molecular Sciences 22:17, pages 9187.
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Josine M de Winter, Charlotte Gineste, Elisa Minardi, Lorenza Brocca, Maira Rossi, Tamara Borsboom, Alan H Beggs, Monique Bernard, David Bendahan, Darren T Hwee, Fady I Malik, Maria Antonietta Pellegrino, Roberto Bottinelli, Julien Gondin & Coen A C Ottenheijm. (2021) Acute and chronic tirasemtiv treatment improves in vivo and in vitro muscle performance in actin-based nemaline myopathy mice . Human Molecular Genetics 30:14, pages 1305-1320.
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Martijn van de Locht, Sandra Donkervoort, Josine M. de Winter, Stefan Conijn, Leon Begthel, Benno Kusters, Payam Mohassel, Ying Hu, Livija Medne, Colin Quinn, Steven A. Moore, A. Reghan Foley, Gwimoon Seo, Darren T. Hwee, Fady I. Malik, Thomas Irving, Weikang Ma, Henk L. Granzier, Erik-Jan Kamsteeg, Kalyan Immadisetty, Peter Kekenes-Huskey, José R. Pinto, Nicol Voermans, Carsten G. Bönnemann & Coen A.C. Ottenheijm. (2021) Pathogenic variants in TNNC2 cause congenital myopathy due to an impaired force response to calcium. Journal of Clinical Investigation 131:9.
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O. E. Karpicheva. (2021) Hallmark Features of the Tropomyosin Regulatory Function in Several Variants of Congenital Myopathy. Journal of Evolutionary Biochemistry and Physiology 57:3, pages 646-665.
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Monica X. Li, Pascal Mercier, James J. Hartman & Brian D. Sykes. (2021) Structural Basis of Tirasemtiv Activation of Fast Skeletal Muscle . Journal of Medicinal Chemistry 64:6, pages 3026-3034.
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Silvia Scaricamazza, Illari Salvatori, Alberto Ferri & Cristiana Valle. (2021) Skeletal Muscle in ALS: An Unappreciated Therapeutic Opportunity?. Cells 10:3, pages 525.
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Khulud Alsulami & Steven Marston. (2020) Small Molecules Acting on Myofilaments as Treatments for Heart and Skeletal Muscle Diseases. International Journal of Molecular Sciences 21:24, pages 9599.
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Seward B. Rutkove, Pushpa Narayanaswami, Visar Berisha, Julie Liss, Shira Hahn, Kerisa Shelton, Kristin Qi, Sarbesh Pandeya & Jeremy M. Shefner. (2020) Improved ALS clinical trials through frequent at‐home self‐assessment: a proof of concept study. Annals of Clinical and Translational Neurology 7:7, pages 1148-1157.
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Humberto Foyaca Sibat & Lourdes de Fátima Ibañez Valdés. 2020. Novel Aspects on Motor Neuron Disease. Novel Aspects on Motor Neuron Disease.
Tereza Filipi, Zuzana Hermanova, Jana Tureckova, Ondrej Vanatko & Miroslava Anderova. (2020) Glial Cells—The Strategic Targets in Amyotrophic Lateral Sclerosis Treatment. Journal of Clinical Medicine 9:1, pages 261.
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Eun-Jeong Lee, Justin Kolb, Darren T. Hwee, Fady I. Malik & Henk L. Granzier. (2019) Functional Characterization of the Intact Diaphragm in a Nebulin-Based Nemaline Myopathy (NM) Model-Effects of the Fast Skeletal Muscle Troponin Activator tirasemtiv. International Journal of Molecular Sciences 20:20, pages 5008.
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Lisanne Roesthuis, Hans van der Hoeven, Christer Sinderby, Tim Frenzel, Coen Ottenheijm, Laurent Brochard, Jonne Doorduin & Leo Heunks. (2019) Effects of levosimendan on respiratory muscle function in patients weaning from mechanical ventilation. Intensive Care Medicine 45:10, pages 1372-1381.
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David G. Allen. (2019) Calcium sensitivity and muscle disease. The Journal of Physiology 597:17, pages 4435-4436.
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