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Expert Opinion on Drug Discovery Volume 10, 2015 - Issue 10
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Review

A retrospective review of the progress in amyotrophic lateral sclerosis drug discovery over the last decade and a look at the latest strategies

Abigail DeLoachUniversity of Arkansas for Medical Sciences, Department of Neurobiology and Developmental Sciences, Little Rock, AR 72205, USA
,
Michael CozartUniversity of Arkansas for Medical Sciences, Department of Pharmacology and Toxicology, Little Rock, AR 72205, USA
,
Arianna KiaeiUniversity of Arkansas for Medical Sciences, Department of Neurobiology and Developmental Sciences, Little Rock, AR 72205, USA
&
Mahmoud KiaeiUniversity of Arkansas for Medical Sciences, Department of Neurobiology and Developmental Sciences, Little Rock, AR 72205, USA;University of Arkansas for Medical Sciences, Department of Pharmacology and Toxicology, Little Rock, AR 72205, USA;University of Arkansas for Medical Sciences, Department of Neurology, 4301 W. Markham St, 846, Little Rock, AR 72205 7199, [email protected]
, PhD
Pages 1099-1118 | Published online: 26 Aug 2015

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Robert L. Sufit, Senda Ajroud-Driss, Patricia Casey & John A. Kessler. (2017) Open label study to assess the safety of VM202 in subjects with amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 18:3-4, pages 269-278.
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Ana Martinez, Maria del Valle Palomo Ruiz, Daniel I. Perez & Carmen Gil. (2017) Drugs in clinical development for the treatment of amyotrophic lateral sclerosis. Expert Opinion on Investigational Drugs 26:4, pages 403-414.
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Articles from other publishers (24)

Alexander Trofimov, Dmitrii Pavlov, Anand Goswami, Anna Gorlova, Kirill Chaprov, Aleksei Umriukhin, Allan Kalueff, Alexey Deykin, Klaus-Peter Lesch, Daniel Anthony & Tatyana Strekalova. (2023) Lipopolysaccharide triggers exacerbated microglial activation, excessive cytokine release and behavioural disturbances in mice with truncated Fused-in-Sarcoma Protein (FUS). Brain, Behavior, & Immunity - Health, pages 100686.
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Jing-Xin Ren, Qian Gao, Xiao-Chao Zhou, Lei Chen, Wei Guo, Kai-Yan Feng, Lin Lu, Tao Huang & Yu-Dong Cai. (2023) Identification of Gene Markers Associated with COVID-19 Severity and Recovery in Different Immune Cell Subtypes. Biology 12:7, pages 947.
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Mohamed F. Elmansy, Cory T. Reidl, Mizzanoor Rahaman, P. Hande Özdinler & Richard B. Silverman. (2023) Small molecules targeting different cellular pathologies for the treatment of amyotrophic lateral sclerosis. Medicinal Research Reviews.
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Rawan Al‐kharboosh, Jonathan Jude Perera, Alexandra Bechtle, Guojun Bu & Alfredo Quinones‐Hinojosa. (2022) Emerging point‐of‐care autologous cellular therapy using adipose‐derived stromal vascular fraction for neurodegenerative diseases. Clinical and Translational Medicine 12:12.
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Lily Kiaei & Mahmoud Kiaei. (2021) RNA as a source of biomarkers for amyotrophic lateral sclerosis. Metabolic Brain Disease 37:6, pages 1697-1702.
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William M. Pardridge. (2022) A Historical Review of Brain Drug Delivery. Pharmaceutics 14:6, pages 1283.
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Yishan Fu, Fei Pan, Lei Zhao, Shuai Zhao, Junjie Yi & Shengbao Cai. (2022) Interfering effects on the bioactivities of several key proteins of COVID-19/variants in diabetes by compounds from Lianqiao leaves: In silico and in vitro analyses. International Journal of Biological Macromolecules 207, pages 715-729.
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Yishan Fu, Yijia Jia, Yilin Sun, Xiaojing Liu, Junjie Yi & Shengbao Cai. (2022) Dietary Flavonoids Alleviate Inflammation and Vascular Endothelial Barrier Dysfunction Induced by Advanced Glycation End Products In Vitro. Nutrients 14:5, pages 1026.
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Agnes Badu-Mensah, Xiufang Guo, Christopher W. McAleer, John W. Rumsey & James J. Hickman. (2020) Functional skeletal muscle model derived from SOD1-mutant ALS patient iPSCs recapitulates hallmarks of disease progression. Scientific Reports 10:1.
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Jayden A. Smith, Alice Braga, Regan Hamel, Carola Rutigliani, Bryan Yu, Luca Peruzzotti-Jametti & Stefano Pluchino. 2020. Handbook of Innovations in Central Nervous System Regenerative Medicine. Handbook of Innovations in Central Nervous System Regenerative Medicine 147 258 .
Klara Valko & Lukasz Ciesla. 2019. 63 117 .
Vijay Kumar, Tara Kashav & Md. Imtaiyaz Hassan. 2019. Pathology, Prevention and Therapeutics of Neurodegenerative Disease. Pathology, Prevention and Therapeutics of Neurodegenerative Disease 207 224 .
Teodorico C. Ramalho, Alexandre A. de Castro, Tássia S. Tavares, Maria C. Silva, Daniela R. Silva, Pedro H. Cesar, Lucas A. Santos, Elaine F.F. da Cunha, Eugenie Nepovimova & Kamil Kuca. (2018) Insights into the pharmaceuticals and mechanisms of neurological orphan diseases: Current Status and future expectations. Progress in Neurobiology 169, pages 135-157.
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Mahmoud Kiaei, Meenakshisundaram Balasubramaniam, Vivek Govind Kumar, Robert J. Shmookler Reis, Mahmoud Moradi & Kottayil I. Varughese. (2018) ALS-causing mutations in profilin-1 alter its conformational dynamics: A computational approach to explain propensity for aggregation. Scientific Reports 8:1.
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Tara Kashav & Vijay Kumar. 2018. Systems Biology. Systems Biology 59 75 .
Bor Luen Tang. (2017) The use of mesenchymal stem cells (MSCs) for amyotrophic lateral sclerosis (ALS) therapy – a perspective on cell biological mechanisms. Reviews in the Neurosciences 28:7, pages 725-738.
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Wei Sun, Wei Zheng & Anton Simeonov. (2017) Drug discovery and development for rare genetic disorders. American Journal of Medical Genetics Part A 173:9, pages 2307-2322.
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Dmitry Petrov, Colin Mansfield, Alain Moussy & Olivier Hermine. (2017) ALS Clinical Trials Review: 20 Years of Failure. Are We Any Closer to Registering a New Treatment?. Frontiers in Aging Neuroscience 9.
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BorLuen Tang. (2017) Amyotrophic lateral sclerosis disease modifying therapeutics: a cell biological perspective. Neural Regeneration Research 12:3, pages 407.
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Mark Kindy, Paul Lupinacci, Raymond Chau, Tony Shum & Dorothy Ko. (2017) A Phase 2A randomized, double-blind, placebo-controlled pilot trial of GM604 in patients with Amyotrophic Lateral Sclerosis (ALS Protocol GALS-001) and a single compassionate patient treatment (Protocol GALS-C). F1000Research 6, pages 230.
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Stephanie Willerth. 2017. Engineering Neural Tissue from Stem Cells. Engineering Neural Tissue from Stem Cells 1 15 .
Vijay Kumar, Asimul Islam, Md. Imtaiyaz Hassan & Faizan Ahmad. (2016) Therapeutic progress in amyotrophic lateral sclerosis-beginning to learning. European Journal of Medicinal Chemistry 121, pages 903-917.
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Mohammad A. Esmaeili, Shilpi Yadav, Ravi Kr. Gupta, Garrett R. Waggoner, Abigail Deloach, Noel Y. Calingasan, M. Flint Beal & Mahmoud Kiaei. (2016) Preferential PPAR-α activation reduces neuroinflammation, and blocks neurodegeneration in vivo . Human Molecular Genetics 25:2, pages 317-327.
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