Jeremy M . Shefner, MD, PhD
Professor
Department
Neurology (Phoenix)
Position
Professor
General
- Brooks Benjamin Rix, Shefner Jeremy M, Jorgenson James A, Newhouse Barbara J, 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 - Muscle & nerve
Berry James D., Paganoni Sabrina, Garret Mark A., Ladha Shaffeeq S., Tsou Amy Y., Provenzano Frank A., Babu Suma, Sherman Alex V., Harris Brent T., Mohan Praveena, Igne Courtney, Bowser Robert, Miller Timothy M., Harms Matthew B., Shneider Neil, Andrews Jinsy, Shefner Jeremy, Phatnani Hemali P., , Access for ALL in ALS: A large-scale, inclusive, collaborative consortium to unlock the molecular and genetic mechanisms of amyotrophic lateral sclerosis
70:6, p. 1140 - 1150 2024 - Amyotrophic lateral sclerosis and frontotemporal degeneration
Stegmann Gabriela, Berisha Visar, Liss Julie, Krantsevich Chelsea, Charles Sherman, Bartlett Meredith, Talkar Tanya, Shefner Jeremy, Rutkove Seward, Kawabata Kan, Automated speech analytics in ALS: higher sensitivity of digital articulatory precision over the ALSFRS-R
25:7-8, p. 767 - 775 2024 - Annals of neurology
Shefner Jeremy M, Cudkowicz Merit E, Failures to Replicate: What Recent Negative Phase 3 Trials Have Taught Us about Amyotrophic Lateral Sclerosis Clinical Research
96:2, p. 211 - 215 2024 - Lancet neurology
Benatar Michael, Morales Raul Juntas, Camu William, Kuzma-Kozakiewicz Magdalena, van den Berg Leonard H, Chio Adriano, Blaettler Thomas, Shefner Jeremy M, Hansen Thomas, Rom Dror, Geist Marie A, , Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial
23:7, p. 687 - 699 2024 - Amyotrophic lateral sclerosis and frontotemporal degeneration
Genge Angela, McDermott Chris, Chio Adriano, Al-Chalabi Ammar, Van Damme Philip, Glass Jonathan, Cedarbaum Jesse M., Berry James, van Eijk Ruben P.A., Shefner Jeremy, , The ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials
25:3-4, p. 382 - 387 2024 - Neurology
Katz Jonathan S., Sierra Rafael, Vu Tuan, Chio Adriano, McDermott Christopher, Devos David, Johnston Mayzie, Shefner Jeremy M., Tansy Aaron, Trimmer Jeff, CARDINALS: A Phase 2, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Utreloxastat (PTC857) in Patients with Amyotrophic Lateral Sclerosis (P5-11.010)
102:7_supplement_1 2024 - Neurology
Shefner Jeremy, Leitner Melanie, Oskarsson Bjorn, Cohen Yael, Chen Kelly, Berry James, Macklin Eric, Goldberg Paul, Pothier Lindsay, Geva Michal, Chibnik Lori, Hayden Michael, Cudkowicz Merit, Paganoni Sabrina, Pridopidine for the Treatment of ALS—Results from the Phase 2 Healey ALS Platform Trial (S5.003)
102:7_supplement_1 2024 - Annals of neurology
Benatar Michael, Shefner Jeremy, Ostrow Lyle W., Lewcock Joseph W, Bennett Frank, Larkin Paul, Bowser Robert, Bruijn Lucie, Wuu Joanne, Biomarker Qualification for Neurofilament Light Chain in Amyotrophic Lateral Sclerosis: Theory and Practice
95:2, p. 211 - 216 2024 - Amyotrophic lateral sclerosis and frontotemporal degeneration
Shefner Jeremy M., Kittle Gale, Bunte Tommy, Genge Angela, van den Berg Leonard H., Harmonized standard operating procedures for administering the ALS functional rating scale-revised
25:1-2, p. 26 - 33 2024 - 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 - Annals of neurology
Quintana Melanie, Yu Hong, Drake Kristin, Cudkowicz Merit, Paganoni Sabrina, Macklin Eric A., Chase Marianne, Andrews Jinsy, Sherman Alexander V., Berry James D., Saville Benjamin R., Vestrucci Matteo, Detry Michelle A., Chibnik Lori, Shefner Jeremy, , Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis
94:3, p. 547 - 560 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 - Amyotrophic lateral sclerosis and frontotemporal degeneration
Salomon-Zimri Shiran, Ennist David L., Elgrart Katya, Beaulieu Danielle, Berry James D., Drory Vivian E., Birman Nurit, Pushett Avital, Russek-Blum Niva, Van Eijk Ruben P.A., Eitan Erez, Paganoni Sabrina, Shefner Jeremy M., Abramovich Beatrice, Combination of ciprofloxacin/celecoxib as a novel therapeutic strategy for ALS
24:3-4, p. 263 - 271 2023 - Amyotrophic lateral sclerosis and frontotemporal degeneration
Hamilton Jenny, Mohan Praveena, Kittle Gale, Shefner Jeremy M., Impact of mode of training and recertification on ALSFRS-R rater performance
24:3-4, p. 289 - 294 2023 - Amyotrophic lateral sclerosis and frontotemporal degeneration
Gebrehiwet Paulos, Sarocco Phil, Wei Jenny, Meng Lisa, Rudnicki Stacy A., Genge Angela, Chiò Adriano, Andrews Jinsy A., Wolff Andrew A., Jackson Carlayne E., Lechtzin Noah, Miller Timothy M., Shefner Jeremy M., MiToS and King’s staging as clinical outcome measures in ALS: a retrospective analysis of the FORTITUDE-ALS trial
24:3-4, p. 304 - 310 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 - JAMA neurology
Shefner Jeremy M., Maragakis Nicholas J., Bedlack Richard, Andrews Jinsy A., Berry James D., Bowser Robert, Brown Robert, Glass Jonathan D., Miller Timothy M., Rothstein Jeffrey D., Cudkowicz Merit E., Amyotrophic Lateral Sclerosis Clinical Trials and Interpretation of Functional End Points and Fluid Biomarkers: A Review
79:12, p. 1312 - 1318 2022 - Muscle & nerve
Shefner Jeremy M., Changing amyotrophic lateral sclerosis diagnostic criteria: Will it impact clinical trials?
66:4, p. 377 - 379 2022 - Journal of neurology, neurosurgery and psychiatry
Paganoni Sabrina, Hendrix Suzanne, Elliott Michael A., Dickson Samuel P., Knowlton Newman, Berry James D., Owegi Margaret Ayo, Maiser Samuel, Karam Chafic, Caress James B., Shefner Jeremy M., , Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial
93:8, p. 871 - 875 2022 - BMJ neurology open
Hannaford Andrew, Vucic Steve, Geevasinga Nimesh, Menon Parvathi, Shefner Jeremy, Kiernan Matthew, Pavey Nathan, Bos Mehdi Van den, 2277 Diagnostic utility of gold coast criteria in amyotrophic lateral sclerosis
4:Suppl 1, p. A3 - A3 2022 - Muscle & nerve
Miller Robert G., McGrath Michael S., Zhang Rongzhen, Bracci Paige M., Azhir Ari, Barohn Richard, Bedlack Richard, Benatar Michael, Berry James D., Cudkowicz Merit, Kasarskis Edward J., Mitsumoto Hiroshi, Manousakis Georgios, Walk David, Oskarsson Bjorn, 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 - Annals of neurology
Cudkowicz Merit E., Shefner Jeremy M., Regulatory Approval in ALS; When Is a Single Study Enough?
91:6, p. 737 - 739 2022 - Amyotrophic lateral sclerosis and frontotemporal degeneration
Rudnicki Stacy A., Wolff Andrew A., Malik Fady, Meng Lisa, Wei Jenny, Jackson Carlayne, Shefner Jeremy M., Andrews Jinsy A., Genge Angela, Cockroft Bettina M., Lechtzin Noah, Miller Timothy M., , Prescription and acceptance of durable medical equipment in FORTITUDE-ALS, a study of reldesemtiv in ALS: post hoc analyses of a randomized, double-blind, placebo-controlled clinical trial
23:3-4, p. 263 - 270 2022 - Brain (London, England : 1878)
Benatar Michael, Shefner Jeremy, Rosen Howard, Fradette Stephanie, Wuu Joanne, McHutchison Caroline, Postuma Ronald B., Boeve Bradley F., Petersen Ronald, Ross Christopher A., Arias Jalayne J., , Preventing amyotrophic lateral sclerosis: insights from pre-symptomatic neurodegenerative diseases
145:1, p. 27 - 44 2022 - Annals of neurology
Paganoni Sabrina, Shefner Jeremy, Berry James D., Quintana Melanie, Macklin Eric, Saville Benjamin R., Detry Michelle A., Chase Marianne, Sherman Alexander V., Yu Hong, Drake Kristin, , Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development
91:2, p. 165 - 175 2022 - Alzheimer's & dementia : diagnosis, assessment & disease monitoring
Stegmann Gabriela, Shefner Jeremy, Hahn Shira, Bhandari Samarth, Kawabata Kan, Mueller Kimberly, Duncan Cayla Jessica, Liss Julie, Berisha Visar, Automated semantic relevance as an indicator of cognitive decline: Out-of-sample validation on a large-scale longitudinal dataset
14:1, p. e12294 - n/a 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., Cudkowicz Merit E., Hardiman Orla, Cockroft Bettina M., 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, Farwell Wildon, De Vivo Darryl C., Schneider Eugene, Green Allison M., Sun Peng, Bhan Ishir, 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
- Invited Speaker, 2018 Northeast ALS Clinical Trials Consortium Annual Meeting, Clearwater FL. 2018
- Plenary Speaker, 2018 FightMND Australasion ALS Symposium, Melbourne, Australia. 2018
- Lisa Krivickas Lecturer, Massachusetts General Hospital, Boston, MA. 2018
- Invited Speaker, Packard Symposium on ALS, Baltimore, MD. 2017
- Grand Rounds, Houston Methodist Medical Center. 2017
- Grand Rounds, University of Arizona College of Medicine. 2017