Citations 16
Primary lateral sclerosis natural history study – planning, designing, and early enrollment
Mitsumoto, Hiroshi , Jang, Grace E. , Simmons, Zachary , Sherman, Alexander , Heitzman, Daragh, Sorenson, Eric J. , Andrews, Jinsy , Harms, Matthew , Santella, Regina M. , Ajroud‐Driss, Senda , Fernandes, Joseph Americo, Gwathmey, Kelly , Habib, Ali A. , Maragakis, Nicholas J. , Walk, David , Fournier, Christina , Heiman‐Patterson, Terry , Wymer, James , Diaz, Frank , Scelsa, Stephen N. , Elman, Lauren , Genge, Angela , Hayat, Ghazala , Jawdat, Omar, Johnston, Wendy , Joyce, Nanette C. , Kasarskis, Edward J. , Kisanuki, Yaz Y. , Lomen‐Hoerth, Catherine , Pulley, Michael , Shah, Jaimin , Shoesmith, Christen , Zinman, Lorne , Group., The PLS Natural History Study, Lee, Ikjae , Sorenson, Eric, Shah, Jaimin S, Simmons, Zachary , Maragakis, Nicholas , Diaz, Frank , Walk, David , Lee, Ik Jae , Cheung, Ken , Harms, Matthew B. , Shneider, Neil A. , Paganoni, Sabrina , Fernandes, J. Americo, Burke, Katherine M. , Goutman, Stephen A. , Group., The PLS Natural History Study
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration | December 28, 2022 | Volume: 24 | Issue: 5-6 | Pages: 394-404
MN-166 (ibudilast) in Amyotrophic Lateral Sclerosis in a Phase IIb/III Study: COMBAT-ALS Study Design
Oskarsson, Björn , Maragakis, Nicholas J. , Bedlack, Richard , Goyal, Namita , Meyer, Jenny, Genge, Angela , Bodkin, Cynthia, Maiser, Samuel, Staff, Nathan P. , Zinman, Lorne , Olney, Nicholas , Turnbull, John , Klonowski, Emelia, Makhay, Malath, Yasui, Seiichi , Matsuda, Kazuko, Maragakis, Nicholas , Bedlack, Richard S , Staff, Nathan P , Brooks, Benjamin Rix , Yasui, Seiichi , Oskarsson, Björn , Goyal, Namita A , Brooks, Benjamin Rix
Neurodegenerative Disease Management | November 24, 2021 | Volume: 11 | Issue: 6 | Pages: 431-443
Association of Variants in the SPTLC1 Gene With Juvenile Amyotrophic Lateral Sclerosis
Johnson, Janel O., Chia, Ruth , Miller, Danny E. , Kumaran, Ravindran, Abramzon, Yevgeniya, Alahmady, Nada F. , Topp, Simon , Gibbs, J. Raphael , Cookson, Mark , Sabir, Marya S. , Dalgard, Clifton L. , Troakes, Claire , Jones, Ashley R., Shatunov, Aleksey , Iacoangeli, Alfredo , Khleifat, Ahmad Al , Ticozzi, Nicola , Gellera, Cinzia , Blair, Ian P. , Dobson‐Stone, Carol , Bonkowski, Emily , Palvadeau, Robin , Tienari, Pentti J. , Morrison, Karen , Shaw, Pamela J. , Brown, Robert H., Calvo, Andrea, Al-Saif, Hind, Leigh, Fawn, Chang, Irene J. , Perlman, Seth J. , Scott, Anna I. , Başak, A. 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JAMA Neurology | August 30, 2021 | Volume: 78 | Issue: 10 | Pages: 1236
Effect of Ezogabine on Cortical and Spinal Motor Neuron Excitability in Amyotrophic Lateral Sclerosis
Wainger, Brian J. , McIlduff, Courtney, Maragakis, Nicholas J. , Bedlack, Richard , Goyal, Namita , Rutkove, Seward B. , Lange, Dale J. , Rivner, Michael H. , Goutman, Stephen A. , Mauricio, Elizabeth A. , Baloh, Robert H. , Simmons, Zachary , Pothier, Lindsay , Kassis, Sylvia Baedorf , La, Thuong, Hall, Meghan, Evora, Armineuza, Klements, David , Hurtado, Aura, Pereira, João D. , Koh, Joan, Celnik, Pablo , Chaudhry, Vinay , Gable, Karissa , Juel, Vern C. , Phielipp, Nicolás , Marei, Adel, Rosenquist, Peter B. , Oskarsson, Björn , Lewis, Richard A. , Kaur, Divpreet, Kiskinis, Evangelos , Eggan, Kevin , Weiss, Michael D. , David, William S., Davila-Pérez, Paula , Camprodon, Joan A. , Kiernan, Matthew C. , Atassi, Nazem , Cudkowicz, Merit , Goutman, Stephen A , Simmons, Zachary , Oskarsson, Björn , Maragakis, Nicholas , Bedlack, Richard S , Cudkowicz, Merit E , Kiernan, Matthew C , Macklin, Eric A. , Vucic, Steve , Paganoni, Sabrina , Ladha, Shafeeq , Mauricio, Elizabeth A. , Puerto, Aura Maria Hurtado , Meehan, Sean K. , Woolf, Clifford J. , Berry, James D. , Pascual‐Leone, Álvaro , Shefner, Jeremy M.
JAMA Neurology | November 23, 2020 | Volume: 78 | Issue: 2 | Pages: 186
Addressing heterogeneity in amyotrophic lateral sclerosis CLINICAL TRIALS
Goyal, Namita , Windebank, Anthony J. , Staff, Nathan P. , Maragakis, Nicholas J. , Berg, Leonard H. van den , Genge, Angela , Baloh, Robert H. , Kern, R. , Gothelf, Yaël, Lebovits, Chaim, Cudkowicz, Merit , Windebank, Anthony, Staff, Nathan P , Maragakis, Nicholas , Cudkowicz, Merit E , Berry, James D. , Miller, Robert G.
Muscle & Nerve | January 3, 2020 | Volume: 62 | Issue: 2 | Pages: 156-166
Abstract Amyotrophic lateral sclerosis (ALS) is a debilitating neurodegenerative disorder with complex biology and significant clinical heterogeneity. Many preclinical and early phase ALS clinical trials have yielded promising results that could not be replicated in larger phase 3 confirmatory trials. One reason for the lack of reproducibility may be ALS biological and clinical heterogeneity. Therefore, in this review, we explore sources of ALS heterogeneity that may reduce statistical power to evaluate efficacy in ALS trials. We also review efforts to manage clinical heterogeneity, including use of validated disease outcome measures, predictive biomarkers of disease progression, and individual clinical risk stratification. We propose that personalized prognostic models with use of predictive biomarkers may identify patients with ALS for whom a specific therapeutic strategy may be expected to be more successful. Finally, the rapid application of emerging clinical and biomarker strategies may reduce heterogeneity, increase trial efficiency, and, in turn, accelerate ALS drug development.
Primary lateral sclerosis (PLS) functional rating scale: PLS‐specific clinimetric scale
Mitsumoto, Hiroshi , Chiuzan, Codruța , Gilmore, Madison , Zhang, Yuan , Simmons, Zachary , Kisanuki, Yasushi, Zinman, Lorne , Jawdat, Omar, Sorenson, Eric J. , Floeter, Mary Kay, Filho, J. Americo M. Fernandes, Heitzman, Daragh, Fournier, Christina , Oskarsson, Björn , Heiman‐Patterson, Terry , Maragakis, Nicholas J. , Joyce, Nanette C. , Hayat, Ghazala , Nations, Sharon , Scelsa, Stephen N. , Walk, David , Elman, Lauren , Hupf, Jonathan , McHale, Brittany , group, the PLSFRS study, Sorenson, Eric, Simmons, Zachary , Oskarsson, Björn , Maragakis, Nicholas , Walk, David , Paganoni, Sabrina , Pioro, Erik P. , group, the PLSFRS study
Muscle & Nerve | November 23, 2019 | Volume: 61 | Issue: 2 | Pages: 163-172
AbstractIntroductionOur research aim was to develop a novel clinimetric scale sensitive enough to detect disease progression in primary lateral sclerosis (PLS).MethodsA prototype of the PLS Functional Rating Scale (PLSFRS) was generated. Seventy‐seven participants with PLS were enrolled and evaluated at 21 sites that comprised the PLSFRS study group. Participants were assessed using the PLSFRS, Neuro‐Quality of Life (QoL), Schwab‐England Activities of Daily Living (ADL), and the Clinical Global Impression of Change scales. Participants completed telephone assessments at 12, 24, and 48 weeks after enrollment.ResultsThe PLSFRS demonstrated internal consistency as well as intrarater, interrater, telephone test‐retest reliability, and construct validity. Significant changes in disease progression were detected at 6 and 12 months; changes measured by the PLSFRS vs the ALSFRS‐R were significantly higher.DiscussionThe PLSFRS is a valid tool to assess the natural history of PLS in a shorter study period.