Brooks Benjamin Rix, Jorgenson James A, Newhouse Barbara J, Shefner Jeremy M, Agnese Wendy, Edaravone in the treatment of amyotrophic lateral sclerosis: efficacy and access to therapy - a roundtable discussion 2018
Articles
Handbook of Clinical Neurology Andrews Jinsy A., Shefner Jeremy M., Clinical neurophysiology of anterior horn cell disorders [Book Chapter] 2019
Publications
Amyotrophic lateral sclerosis and frontotemporal degeneration Rudnicki Stacy A., Wei Jenny, Gebrehiwet Paulos, Kupfer Stuart, Malik Fady I., Meng Lisa, Simkins Tyrell, Wolff Andrew A., Shefner Jeremy M., Participant, site personnel and sponsor perspectives on decentralized trial features in COURAGE-ALS: a randomized clinical trial 26:7-8, p. 812 - 820 2025
Journal of neuromuscular diseases Simkins (lead) Tyrell, Kupfer Stuart, Shefner Jeremy M, Malik Fady I, van Eijk Ruben PA, Meng Lisa, Rudnicki Stacy A, Wei Jenny, Application of the ENCALS predictive survival model in assessing the effect of the 24/44 inclusion criteria in FORTITUDE-ALS 12:5, p. 679 - 682 2025
Lancet neurology Ladha Shafeeq, Wang Jianing, Chibnik Lori B., Quintana Melanie, Macklin Eric A., Saville Benjamin R., Detry Michelle A., Vestrucci Matteo, Paulon Giorgio, McGlothlin Anna, Shefner Jeremy, , Safety and efficacy of trehalose in amyotrophic lateral sclerosis (HEALEY ALS Platform Trial): an adaptive, phase 2/3, double-blind, randomised, placebo-controlled trial 24:6, p. 500 - 511 2025
JAMA neurology Shefner Jeremy M., Andrews Jinsy A., Cudkowicz Merit E., Genge Angela, Hardiman Orla, Al-Chalabi Ammar, Chio Adriano, Corcia Philippe, Couratier Philippe, De Carvalho Mamede, , Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial 82:5, p. 477 - 485 2025
Amyotrophic lateral sclerosis and frontotemporal degeneration Simkins Tyrell J., Rudnicki Stacy A., Kupfer Stuart, Malik Fady I., Meng Lisa, Bowser Robert, Wei Jenny, Shefner Jeremy M., Plasma neurofilament analysis in VITALITY-ALS 26:1-2, p. 103 - 112 2025
Amyotrophic lateral sclerosis and frontotemporal degeneration Rudnicki Stacy A., Corcia Philippe, Al-Chalabi Ammar, Andrews Jinsy A., Chio Adriano, Genge Angela, Couratier Philippe, Cudkowicz Merit E., De Carvalho Mamede, Hardiman Orla, Shefner Jeremy M., , Hospitalizations as an outcome measure in COURAGE-ALS 26:7-8, p. 802 - 811 2025
Amyotrophic lateral sclerosis and frontotemporal degeneration Shefner Jeremy M, Kupfer Stuart, Jacobsen Bill, Wolff Andrew A, Malik Fady I, Meng Lisa, Wei Jenny, Rudnicki Stacy A, Relationship between quantitative strength and functional outcomes in the phase 2 FORTITUDE-ALS trial 25:1-2, p. 162 - 169 2024
Brain (London, England : 1878) Shefner Jeremy, Musaro Antonio, Robitaille Richard, de Carvalho Mamede, Rutkove Seward, Ludolph Albert, Dupuis Luc, Ngo Shyuan, Lunetta Christian, Steyn Frederik, Skeletal muscle in amyotrophic lateral sclerosis 146:11, p. 4425 - 4436 2023
Amyotrophic lateral sclerosis and frontotemporal degeneration Shefner Jeremy M., Couratier Philippe, Cudkowicz Merit E., Genge Angela, De Carvalho Mamede, Al-Chalabi Ammar, Cockroft Bettina M., Corcia Philippe, Andrews Jinsy A., Chio Adriano, , COURAGE-ALS: a randomized, double-blind phase 3 study designed to improve participant experience and increase the probability of success 24:5-6, p. 523 - 534 2023
Journal of medical economics Gebrehiwet Paulos, Miller Timothy M., Shefner Jeremy M., Sarocco Phil, Meng Lisa, Rudnicki Stacy A., Wei Jenny, Wolff Andrew A., Butzner Michael, Hughes Dyfrig A., Chiò Adriano, Andrews Jinsy A., Genge Angela, Jackson Carlayne E., Lechtzin Noah, Health utilities and quality-adjusted life years for patients with amyotrophic lateral sclerosis receiving reldesemtiv or placebo in FORTITUDE-ALS 26:1, p. 488 - 493 2023
Muscle & nerve Miller Robert G., Mitsumoto Hiroshi, Zhang Rongzhen, Bracci Paige M., Azhir Ari, Barohn Richard, Bedlack Richard, Benatar Michael, Berry James D., Cudkowicz Merit, Kasarskis Edward J., Manousakis Georgios, Walk David, Oskarsson Bjorn, McGrath Michael S., Shefner Jeremy, Phase 2B randomized controlled trial of NP001 in amyotrophic lateral sclerosis: Pre-specified and post hoc analyses 66:1, p. 39 - 49 2022
Amyotrophic lateral sclerosis and frontotemporal degeneration Stegmann Gabriela M., Liss Julie, Hahn Shira, Duncan Cayla J., Rutkove Seward B., Shefner Jeremy M., Berisha Visar, Estimation of forced vital capacity using speech acoustics in patients with ALS 22:S1, p. 14 - 21 2021
Nature reviews. Neurology Kiernan Matthew C., Talbot Kevin, Vucic Steve, Al-Chalabi Ammar, McDermott Christopher J., Hardiman Orla, Shefner Jeremy M., Van den Berg Leonard H., Huynh William, Cudkowicz Merit, Talman Paul, Dharmadasa Thanuja, Wicks Paul, Reilly Claire, Turner Martin R., Improving clinical trial outcomes in amyotrophic lateral sclerosis 17:2, p. 104 - 118 2021
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration Shefner Jeremy M., Malik Fady I., Andrews Jinsy A., Meng Lisa, Rudnicki Stacy A., Wolff Andrew A., Cockroft Bettina M., Cudkowicz Merit E., Hardiman Orla, Lee Jacqueline H., A phase III trial of tirasemtiv as a potential treatment for amyotrophic lateral sclerosis 20:7-8, p. 584 - 594 2019
Neurology Andrews Jinsy A., Bruijn Lucie I., Shefner Jeremy M., ALS drug development guidances and trial guidelines: Consensus and opportunities for alignment 93:2, p. 66 - 71 2019
Neurology Darras Basil T., Montes Jacqueline, Chiriboga Claudia A., Iannaccone Susan T., Swoboda Kathryn J., Mignon Laurence, Xia Shuting, Bennett C. Frank, Bishop Kathie M., Shefner Jeremy M., Gheuens Sarah, De Vivo Darryl C., Green Allison M., Sun Peng, Bhan Ishir, Schneider Eugene, Farwell Wildon, Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies 92:21, p. e2492 - e2506 2019
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration Rutkove Seward B., Qi Kristin, Liss Julie, Berisha Visar, Shefner Jeremy M., Shelton Kerisa, ALS longitudinal studies with frequent data collection at home: study design and baseline data 20:1-2, p. 61 - 67 2019
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration Shefner Jeremy M., Rutkove Seward B., Caress James B., Benatar Michael, David William S., Cartwright Michael S., Macklin Eric A., Bohorquez Jose L., Reducing sample size requirements for future ALS clinical trials with a dedicated electrical impedance myography system 19:7-8, p. 555 - 561 2018
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration Lechtzin Noah, Shefner Jeremy, Van den Berg Leonard H., Andrews Jinsy A., Genge Angela, Cudkowicz Merit E., Mitsumoto Hiroshi, Mora Jesus S., Hardiman Orla, de Carvalho Mamede, Respiratory measures in amyotrophic lateral sclerosis 19:5-6, p. 321 - 330 2018
European Journal of Neurology Smith R. A., Macklin E. A., Myers K. J., Pattee G. L., Goslin K. L., Meekins G. D., Green J. R., Shefner J. M., Pioro E. P., Assessment of bulbar function in amyotrophic lateral sclerosis: validation of a self-report scale (Center for Neurologic Study Bulbar Function Scale) 25:7, p. 907 - e66 2018
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration Andrews Jinsy A., Lee Jacqueline, Cudkowicz Merit E., Hardiman Orla, Meng Lisa, Bian Amy, Wolff Andrew A., Malik Fady I., Shefner Jeremy M., 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 19:3-4, p. 259 - 266 2018
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration Meng Lisa, Wolff Andrew, Shefner Jeremy M., Andrews Jinsy, Jordan Scott, Bian Amy, Profile of medical care costs in patients with amyotrophic lateral sclerosis in the Medicare programme and under commercial insurance 19:1-2, p. 134 - 142 2018
JAMA Neurology Andrews Jinsy A., Bozik Michael E., Kulke Sarah F., Rudnicki Stacy A., Wolff Andrew A., Malik Fady I., Shefner Jeremy M., Meng Lisa, Association Between Decline in Slow Vital Capacity and Respiratory Insufficiency, Use of Assisted Ventilation, Tracheostomy, or Death in Patients With Amyotrophic Lateral Sclerosis 75:1, p. 58 - 58 2018
Presentations
Invited Speaker, 2019 California ALS Summit, Irvine CA. 2019