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Study design

Improving efficiency of ALS clinical trials using lead‐in designs

Pages 57-60 | Published online: 10 Jul 2009

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Rosario Vasta, Luca Solero, Francesca Palumbo, Umberto Manera, Maria Claudia Torrieri, Maurizio Grassano, Antonio Canosa, Cristina Moglia, Andrea Calvo & Adriano Chiò. (2022) Can amyotrophic lateral sclerosis progression really pause? A cohort study using the medical research council scale. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 23:5-6, pages 383-389.
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Alon Abraham, Beatrice Nefussy, Yaara Fainmesser, Yoram Ebrahimi, Arnon Karni & Vivian E. Drory. (2019) Early post-marketing experience with edaravone in an unselected group of patients with ALS. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 20:3-4, pages 260-263.
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Yu Zhang, Norbert Schuff, Susan C. Woolley, Gloria C. Chiang, Lauren Boreta, Joel Laxamana, Jonathan S. Katz & Michael W. Weiner. (2011) Progression of white matter degeneration in amyotrophic lateral sclerosis: A diffusion tensor imaging study. Amyotrophic Lateral Sclerosis 12:6, pages 421-429.
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Benjamin C. Cheah, Steve Vucic, Arun V. Krishnan, Robert A. Boland & Matthew C. Kiernan. (2011) Neurophysiological index as a biomarker for ALS progression: Validity of mixed effects models. Amyotrophic Lateral Sclerosis 12:1, pages 33-38.
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David A. Schoenfeld & Merit Cudkowicz. (2008) Design of phase II ALS clinical trials. Amyotrophic Lateral Sclerosis 9:1, pages 16-23.
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Hua Wang, Kylyana S. Larriviere, Kristen E. Keller, Kathleen A. Ware, Ted M. Burns, Mark A. Conaway, David Lacomis, Gary L. Pattee, Lawrence H. Phillips$suffix/text()$suffix/text(), Nina J. Solenski, Sasa A. Zivkovic & James P. Bennett$suffix/text()$suffix/text(). (2008) R(+) pramipexole as a mitochondrially focused neuroprotectant: Initial early phase studies in ALS. Amyotrophic Lateral Sclerosis 9:1, pages 50-58.
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Swati Dhasmana, Anupam Dhasmana, Acharan S. Narula, Meena Jaggi, Murali M. Yallapu & Subhash C. Chauhan. (2022) The panoramic view of amyotrophic lateral sclerosis: A fatal intricate neurological disorder. Life Sciences 288, pages 120156.
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Matthew C. Kiernan, Steve Vucic, Kevin Talbot, Christopher J. McDermott, Orla Hardiman, Jeremy M. Shefner, Ammar Al-Chalabi, William Huynh, Merit Cudkowicz, Paul Talman, Leonard H. Van den Berg, Thanuja Dharmadasa, Paul Wicks, Claire Reilly & Martin R. Turner. (2020) Improving clinical trial outcomes in amyotrophic lateral sclerosis. Nature Reviews Neurology 17:2, pages 104-118.
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R. Vasta, F. D'Ovidio, A. Canosa, U. Manera, M. C. Torrieri, M. Grassano, F. De Marchi, L. Mazzini, C. Moglia, A. Calvo & A. Chiò. (2020) Plateaus in amyotrophic lateral sclerosis progression: results from a population‐based cohort. European Journal of Neurology 27:8, pages 1397-1404.
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Alexander G Thompson, Elizabeth Gray, Alexander Bampton, Dominika Raciborska, Kevin Talbot & Martin R Turner. (2019) CSF chitinase proteins in amyotrophic lateral sclerosis. Journal of Neurology, Neurosurgery & Psychiatry 90:11, pages 1215-1220.
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Stephanie R. ShepheardJoanne WuuMichell CardosoLuke WiklendtPhil G. DinningTim ChatawayDavid SchultzMichael BenatarMary-Louise Rogers. (2017) Urinary p75 ECD . Neurology 88:12, pages 1137-1143.
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Dan H Moore, Jonathan S Katz & Robert G Miller. (2011) A review of clinical trial designs in amyotrophic lateral sclerosis. Neurodegenerative Disease Management 1:6, pages 481-490.
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Adriano Chiò, Gabriele Mora, Vincenzo La Bella, Claudia Caponnetto, Gianluigi Mancardi, Mario Sabatelli, Gabriele Siciliano, Vincenzo Silani, Massimo Corbo, Cristina Moglia, Andrea Calvo, Roberto Mutani, Sergio Rutella, Francesca Gualandi, Mario Melazzini, Rosanna Scimè, Mario Petrini, Paola Bondesan, Silvia Garbelli, Stefania Mantovani, Caterina Bendotti & Corrado Tarella. (2011) Repeated courses of granulocyte colony-stimulating factor in amyotrophic lateral sclerosis: Clinical and biological results from a prospective multicenter study. Muscle & Nerve 43:2, pages 189-195.
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Paul H. Gordon, Bin Cheng, Francois Salachas, Pierre-Francois Pradat, Gaelle Bruneteau, Philippe Corcia, Lucette Lacomblez & Vincent Meininger. (2010) Progression in ALS is not linear but is curvilinear. Journal of Neurology 257:10, pages 1713-1717.
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G. LevyP. KaufmannR. BuchsbaumJ. MontesA. BarsdorfR. ArbingV. BattistaX. ZhouH. MitsumotoB. LevinJ.L.P. Thompson. (2006) A two-stage design for a phase II clinical trial of coenzyme Q10 in ALS. Neurology 66:5, pages 660-663.
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