Goutman, Stephen A
Mayo Clinic
Department of Neurology, University of Michigan, Ann Arbor, Michigan, USA.
Citations 6
Primary Lateral Sclerosis Natural History Study: Primary Lateral Sclerosis Functional Rating Scale and Other Outcomes Assessment
Lee, Ikjae , Jang, Grace E. , Sherman, Alexander , Johnston, Wendy , Fernandes, Joseph Americo, Habib, Ali A. , Maragakis, Nicholas J. , Shah, Jaimin , Zinman, Lorne , Floeter, Mary Kay, Ajroud‐Driss, Senda , Gwathmey, Kelly , Heiman‐Patterson, Terry , Jawdat, Omar, Kasarskis, Edward J. , Kisanuki, Yaz Y. , Wymer, James , Fournier, Christina , Hayat, Ghazala , Heitzman, Daragh, Lomen‐Hoerth, Catherine , Pulley, Michael , Scelsa, Stephen N. , Walk, David , Shoesmith, Christen , Sorenson, Eric J. , Elman, Lauren , Harms, Matthew , Hoover, Benjamin N., Yun, RN ByoungSook, Santella, Regina M. , Mitsumoto, Hiroshi , Group, for the PLS NHS Study, Diaz, Frank , Maragakis, Nicholas , Oskarsson, Björn , Goutman, Stephen A , Sorenson, Eric, Walk, David , Cheung, Ken , Diaz, Frank , Fernandes, J. Americo, Paganoni, Sabrina , Burke, Katherine M. , Oskarsson, Björn , Goutman, Stephen A. , Simmons, Zachary , Harms, Matthew B. , Hoover, Benjamin N., Group, for the PLS NHS Study
Annals of Neurology | September 29, 2025 | Volume: 99 | Issue: 2 | Pages: 418-428
Objective The primary lateral sclerosis ( PLS ) consensus diagnostic criteria and functional rating scale ( PLSFRS ) were recently established to facilitate and optimize future PLS clinical trials. We examined the trajectory of the PLSFRS and other functional outcome measures and biomarkers in the PLS Natural History Study ( PLS NHS ) to understand their performance in this prospective cohort. Methods The PLS NHS is a prospective, longitudinal, multicenter study of people living with PLS in different diagnostic categories: early (disease duration <2 years); probable (2–4 years); and definite PLS (4–15 years). PLSFRS scores and other functional outcome measures were collected at baseline, 3‐, 6‐, 9‐, and 12‐month follow‐up visits. Baseline characteristics were compared between the groups. The slopes of the PLSFRS and other functional outcome measures over 12 months were examined in the overall cohort and subgroups using linear mixed‐effect models. The associations between baseline characteristics and the rate of PLSFRS decline were analyzed with linear regression models. Results A total of 76 participants were included: early (n = 6); probable (n = 26); and definite (n = 44) PLS . Baseline PLSFRS total scores were highest in the early PLS group, followed by the probable and definite PLS groups. In the overall cohort, the PLSFRS total score declined by 0.33 points/month (95% confidence interval [0.27–0.39], adjusted p < 0.05). The rate of decline was steepest in the early PLS group, followed by the probable and definite PLS groups. Baseline neurofilament light chain level was associated with the rate of PLSFRS decline over 1 year ( p = 0.001). Interpretation In PLS, the rate of functional decline, as measured by the PLSFRS total score, is faster during the early phase of the disease. Neurofilament light might serve as a prognostic biomarker in PLS. ANN NEUROL 2026;99:418–428
A revision to the United States national ALS registry’s algorithm to improve Case-Ascertainment
Mehta, Paul , Han, Moon, Punjani, Reshma , Brooks, Benjamin Rix , Oskarsson, Björn , Goutman, Stephen A. , Brooks, Benjamin Rix , Oskarsson, Björn , Goutman, Stephen A , Mehta, Paul , Raymond, Jaime S. , Han, Moon, III, Theodore C. Larson , Berry, James D. , Horton, D. Kevin
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration | October 4, 2022 | Volume: 24 | Issue: 3-4 | Pages: 230-236
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
A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS
Andrews, Jinsy , Genge, Angela , Jackson, Carlayne E. , Miller, Timothy M. , Cockroft, Bettina M., Meng, Lisa , Wei, Jenny , Wolff, Andrew A. , Malik, Fady I. , Bodkin, Cynthia, Dionne, Annie , Fee, Dominic B. , Goutman, Stephen A. , Goyal, Namita , Hardiman, Orla , Hayat, Ghazala , Heiman‐Patterson, Terry , Heitzman, Daragh, Johnston, Wendy , Kiernan, Matthew C. , Korngut, Lawrence , Matte, Geneviève, Mora, Jesús S., Needham, Merrilee , Oskarsson, Björn , Pattee, Gary L. , Pulley, Michael , Quan, Dianna , Rezania, Kourosh , Schellenberg, Kerri , Schultz, David , Shoesmith, Christen , Simmons, Zachary , Statland, Jeffrey , Sultan, Shumaila, Weiss, Michael D. , Whyte-Rayson, Ashley, Wymer, James , Zinman, Lorne , Rudnicki, Stacy A. , Oskarsson, Björn , Schellenberg, Kerri L , Goutman, Stephen A , Simmons, Zachary , Hardiman, Orla , Kiernan, Matthew C , Shefner, Jeremy M. , Lechtzin, Noah , Brooks, Benjamin Rix , Caress, James B. , Henderson, Robert D. , Karam, Chafic , Kolb, Stephen J. , Ladha, Shafeeq , Pioro, Erik P. , Swenson, Andrea , Berg, Leonard H. van den , Vu, Tuan , Vucic, Steve
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration | September 24, 2020 | Volume: 22 | Issue: 3-4 | Pages: 287-299
The NEALS primary lateral sclerosis registry
Chan, James , Thrower, Sara, Staff, Nathan P. , Datta, Neil, Kisanuki, Yaz Y. , Drory, Vivian E., Fournier, Christina , Goutman, Stephen A. , Atassi, Nazem , Group, On behalf of the NEALS PLS Registry Study, Jeon, Maryangel, Caldwell, Sarah, McDonough, Timothy, Gentile, Caroline, Liu, Jianing , Turner, M.F., Denny, Carol, Felice, Kevin J. , Green, Misty, Scarberry, Stephanie, Abu-Saleh, Saad, Nefussy, Beatrice, Hastings, Debbie, Kim, S. K., Swihart, Blake, Arcila-Londono, Ximena , Newman, Daniel S. , Silverman, Michael G. , Genge, Angela , Salmon, Kristiana , Elman, Lauren , McCluskey, Leo, Almasy, Kelly, Goslin, Kimberly, CUMMINGS, A. J., Edwards, Eli K., Rivner, Michael H. , Bouchard, Kristy, Quarles, Brandy, Kwan, Justin , Jaffa, Matthew , Baloh, Robert H. , Allred, Peggy, Walk, David , Maiser, Samuel, Manousakis, Georgios , Ferment, Valerie, Fernandes, J. Americo, Thaisetthawatkul, Pariwat , Heimes, Deborah, Sams, Laura, Kahler, Melissa, Corcoran, Alecia, Larriviere, Daniel G., Chotto, Sadie, Juba, Gracy, Staff, Nathan P , Turner, Michelle, Goutman, Stephen A , Walk, David , Paganoni, Sabrina , Marchi, Fabiola De , Pioro, Erik P. , Group, On behalf of the NEALS PLS Registry Study, Caldwell, Sarah, Mcdonough, Timothy, Turner, Michelle, Denny, C. Dimla, Newman, Daniel S., Silverman, Michael, Gotkine, Marc , Cummings, Arlena, Phillips, Melissa
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration | September 11, 2020 | Volume: 21 | Issue: sup1 | Pages: 74-81
Amyotrophic lateral sclerosis–specific quality of life–short form (ALSSQOL‐SF): A brief, reliable, and valid version of the ALSSQOL‐R
Felgoise, Stephanie H. , Feinberg, Richard A. , Stephens, Helen , Clawson, Lora , Elman, Lauren , Goutman, Stephen A. , McCluskey, Leo, Russell, James W. , Tiryaki, Ezgi , Weiss, Michael D. , Simmons, Zachary , Boylan, Kevin, Goutman, Stephen A , Simmons, Zachary , Barkhaus, Paul E. , Boylan, Khrista , Caress, James B.
Muscle & Nerve | July 20, 2018 | Volume: 58 | Issue: 5 | Pages: 646-654
ABSTRACT Introduction: The Amyotrophic Lateral Sclerosis (ALS)‐Specific Quality of Life instrument and its revised version (ALSSQOL and ALSSQOL‐R) have strong psychometric properties, and have demonstrated research and clinical utility. In this study we aimed to develop a short form (ALSSQOL‐SF) suitable for limited clinic time and patient stamina. Methods: The ALSSQOL‐SF was created using Item Response Theory and confirmatory factor analysis on 389 patients. A cross‐validation sample of 162 patients assessed convergent, divergent, and construct validity of the ALSSQOL‐SF compared with psychosocial and physical functioning measures. Results: The ALSSQOL‐SF consisted of 20 items. Compared with the ALSSQOL‐R, optimal precision was retained, and completion time was reduced from 15–25 minutes to 2–4 minutes. Psychometric properties for the ALSSQOL‐SF and its subscales were strong. Discussion: The ALSSQOL‐SF is a disease‐specific global QOL instrument that has a short administration time suitable for clinical use, and can provide clinically useful, valid information about persons with ALS. Muscle Nerve 58: 646–654, 2018