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Research Article

Safety, tolerability and pharmacodynamics of a skeletal muscle activator in amyotrophic lateral sclerosis

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Pages 430-438 | Received 12 Jan 2012, Accepted 02 Apr 2012, Published online: 16 May 2012

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Jeremy M. Shefner, Jinsy A. Andrews, Angela Genge, Carlayne Jackson, Noah Lechtzin, Timothy M. Miller, Bettina M. Cockroft, Lisa Meng, Jenny Wei, Andrew A. Wolff, Fady I. Malik, Cynthia Bodkin, Benjamin R. Brooks, James Caress, Annie Dionne, Dominic Fee, Stephen A. Goutman, Namita A. Goyal, Orla Hardiman, Ghazala Hayat, Terry Heiman-Patterson, Daragh Heitzman, Robert D. Henderson, Wendy Johnston, Chafic Karam, Matthew C. Kiernan, Stephen J. Kolb, Lawrence Korngut, Shafeeq Ladha, Genevieve Matte, Jesus S. Mora, Merrilee Needham, Bjorn Oskarsson, Gary L. Pattee, Erik P. Pioro, Michael Pulley, Dianna Quan, Kourosh Rezania, Kerri L. Schellenberg, David Schultz, Christen Shoesmith, Zachary Simmons, Jeffrey Statland, Shumaila Sultan, Andrea Swenson, Leonard H. Van Den Berg, Tuan Vu, Steve Vucic, Michael Weiss, Ashley Whyte-Rayson, James Wymer, Lorne Zinman & Stacy A. Rudnicki. (2021) A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 22:3-4, pages 287-299.
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Jinsy A. Andrews, Merit E. Cudkowicz, Orla Hardiman, Lisa Meng, Amy Bian, Jacqueline Lee, Andrew A. Wolff, Fady I. Malik & Jeremy M. Shefner. (2018) VITALITY-ALS, a phase III trial of tirasemtiv, a selective fast skeletal muscle troponin activator, as a potential treatment for patients with amyotrophic lateral sclerosis: study design and baseline characteristics. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 19:3-4, pages 259-266.
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Jeremy M. Shefner, Andrew A. Wolff, Lisa Meng, Amy Bian, Jacqueline Lee, Donna Barragan & Jinsy A. Andrews. (2016) A randomized, placebo-controlled, double-blind phase IIb trial evaluating the safety and efficacy of tirasemtiv in patients with amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 17:5-6, pages 426-435.
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Jeremy M. Shefner & Dragos Mihaila. (2014) Assessment of disease progression and functional benefit in neurodegenerative disease: Can we tell the difference?. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 15:5-6, pages 337-343.
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Jeremy M. Shefner, Mary Lou Watson, Lisa Meng & Andrew A. Wolff. (2013) A study to evaluate safety and tolerability of repeated doses of tirasemtiv in patients with amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 14:7-8, pages 574-581.
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Jeremy M. Shefner, Andrew A. Wolff & Lisa Meng. (2013) The relationship between tirasemtiv serum concentration and functional outcomes in patients with ALS. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 14:7-8, pages 582-585.
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Paul Gordon, Philippe Corcia & Vincent Meininger. (2013) New therapy options for amyotrophic lateral sclerosis. Expert Opinion on Pharmacotherapy 14:14, pages 1907-1917.
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Michael P Bova & Gene G Kinney. (2013) Emerging drug targets in amyotrophic lateral sclerosis. Expert Opinion on Orphan Drugs 1:1, pages 5-20.
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Jeremy M. Shefner. (2016) Strength Testing in Motor Neuron Diseases. Neurotherapeutics 14:1, pages 154-160.
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Jean‐Philippe Loeffler, Gina Picchiarelli, Luc Dupuis & Jose‐Luis Gonzalez De Aguilar. (2016) The Role of Skeletal Muscle in Amyotrophic Lateral Sclerosis. Brain Pathology 26:2, pages 227-236.
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Darren T. Hwee, Adam R. Kennedy, James J. Hartman, Julie Ryans, Nickie Durham, Fady I. Malik & Jeffrey R. Jasper. (2015) The Small-Molecule Fast Skeletal Troponin Activator, CK-2127107, Improves Exercise Tolerance in a Rat Model of Heart Failure. Journal of Pharmacology and Experimental Therapeutics 353:1, pages 159-168.
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Donald B. Sanders, Jeffrey Rosenfeld, Mazen M. Dimachkie, Lisa Meng & Fady I. Malik. (2015) A Double-Blinded, Randomized, Placebo-Controlled Trial to Evaluate Efficacy, Safety, and Tolerability of Single Doses of Tirasemtiv in Patients with Acetylcholine Receptor-Binding Antibody-Positive Myasthenia Gravis. Neurotherapeutics 12:2, pages 455-460.
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Nam-Hee KimMin Oh Lee. (2015) Update of Therapeutic Clinical Trials for Amyotrophic Lateral Sclerosis. Korean Journal of Clinical Neurophysiology 17:1, pages 1.
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Richard Hansen, Khalil G. Saikali, Willis Chou, Alan J. Russell, Michael M. Chen, Vipin Vijayakumar, Randall R. Stoltz, Stephane Baudry, Roger M. Enoka, David J. Morgans, Andrew A. Wolff & Fady I. Malik. (2014) Tirasemtiv amplifies skeletal muscle response to nerve activation in humans. Muscle & Nerve 50:6, pages 925-931.
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Timothy A Bauer, Andrew A Wolff, Alan T Hirsch, Lisa L Meng, Kevin Rogers, Fady I Malik & William R Hiatt. (2014) Effect of tirasemtiv, a selective activator of the fast skeletal muscle troponin complex, in patients with peripheral artery disease. Vascular Medicine 19:4, pages 297-306.
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Darren T. Hwee, Adam Kennedy, Julie Ryans, Alan J. Russell, Zhiheng Jia, Aaron C. Hinken, David J. Morgans, Fady I. Malik & Jeffrey R. Jasper. (2014) Fast Skeletal Muscle Troponin Activator tirasemtiv Increases Muscle Function and Performance in the B6SJL-SOD1G93A ALS Mouse Model. PLoS ONE 9:5, pages e96921.
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Nicole L. Nichols, J. Van Dyke, L. Nashold, I. Satriotomo, M. Suzuki & G.S. Mitchell. (2013) Ventilatory control in ALS. Respiratory Physiology & Neurobiology 189:2, pages 429-437.
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Marie Amitani, Akihiro Asakawa, Haruka Amitani & Akio Inui. (2013) Control of food intake and muscle wasting in cachexia. The International Journal of Biochemistry & Cell Biology 45:10, pages 2179-2185.
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Jessica J. Miciak, Leigh C. Warsing, Maria E. Tibbs, Jeffrey R. JASPER, Sarah B. Jampel, Fady I. Malik, Clarke Tankersley & Kathryn R. Wagner. (2013) Fast skeletal muscle troponin activator in the dy2J muscular dystrophy model. Muscle & Nerve 48:2, pages 279-285.
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Josine Marieke de Winter, Danielle Buck, Carlos Hidalgo, Jeffrey R Jasper, Fady I Malik, Nigel F Clarke, Ger J M Stienen, Michael W Lawlor, Alan H Beggs, Coen A C Ottenheijm & Henk Granzier. (2013) Troponin activator augments muscle force in nemaline myopathy patients with nebulin mutations. Journal of Medical Genetics 50:6, pages 383-392.
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Coen A. C. Ottenheijm, Danielle Buck, Josine M. de Winter, Claudia Ferrara, Nicoletta Piroddi, Chiara Tesi, Jeffrey R. Jasper, Fady I. Malik, Hui Meng, Ger J. M. Stienen, Alan H. Beggs, Siegfried Labeit, Corrado Poggesi, Michael W. Lawlor & Henk Granzier. (2013) Deleting exon 55 from the nebulin gene induces severe muscle weakness in a mouse model for nemaline myopathy. Brain 136:6, pages 1718-1731.
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