Publications by authors named "Jeremy Shefner"

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Pridopidine treatment in ALS: subgroup analyses from the HEALEY ALS Platform trial.
Michal Geva, Y Paul Goldberg, Melanie L Leitner, Andres Cruz-Herranz, Randal Hand, Jeremy M Shefner

Amyotroph Lateral Scler Frontotemporal Degener· December 2025

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Hospitalizations as an outcome measure in COURAGE-ALS.
Stacy A Rudnicki, Ammar Al-Chalabi, Jinsy A Andrews, Adriano Chio, Philippe Corcia, Jeremy M Shefner

Amyotroph Lateral Scler Frontotemporal Degener· November 2025

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Participant, site personnel and sponsor perspectives on decentralized trial features in COURAGE-ALS: a randomized clinical trial.
Stacy A Rudnicki, Paulos Gebrehiwet, Stuart Kupfer, Fady I Malik, Lisa Meng, Jeremy M Shefner

Amyotroph Lateral Scler Frontotemporal Degener· November 2025

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Application of the ENCALS predictive survival model in assessing the effect of the 24/44 inclusion criteria in FORTITUDE-ALS.
Tyrell Simkins Lead, Jeremy M Shefner, Stuart Kupfer, Fady I Malik, Lisa Meng

J Neuromuscul Dis· September 2025

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Operational Development and Launch of an Adaptive Platform Trial in Amyotrophic Lateral Sclerosis: Processes and Learnings From the First Four Regimens of the HEALEY ALS Platform Trial.
Brittney A Harkey, Sofia Distefano, Jaclyn A Pagliaro, Lindsay Heyd, Marianne Chase, Jeremy Shefner

Muscle Nerve· August 2025

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Akt Activation With IPL344 Treatment for Amyotrophic Lateral Sclerosis: First in Human, Open-Label Study.
Marc Gotkine, David A Schoenfeld, Ilana Cohen, Jeremy M Shefner, Yossef Lerner

Muscle Nerve· June 2025

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Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial.
Jeremy M Shefner, Merit E Cudkowicz, Angela Genge, Orla Hardiman, Ammar Al-Chalabi

JAMA Neurol· May 2025

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Verdiperstat in Amyotrophic Lateral Sclerosis: Results From the Randomized HEALEY ALS Platform Trial.

JAMA Neurol· April 2025

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Guidance for clinical management of pathogenic variant carriers at elevated genetic risk for ALS/FTD.
Michael Benatar, Terry D Heiman-Patterson, Johnathan Cooper-Knock, Daniel Brickman, Kaitlin B Casaletto, Jeremy Shefner

J Neurol Neurosurg Psychiatry· March 2025

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Plasma neurofilament analysis in VITALITY-ALS.
Tyrell J Simkins, Stuart Kupfer, Fady I Malik, Lisa Meng, Stacy A Rudnicki, Jeremy M Shefner

Amyotroph Lateral Scler Frontotemporal Degener· February 2025

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Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial.
Sabrina Paganoni, Christina N Fournier, Eric A Macklin, Lori B Chibnik, Melanie Quintana, Jeremy M Shefner

JAMA Netw Open· February 2025

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Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial.

JAMA· February 2025

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CNM-Au8 in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial.

JAMA· February 2025

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Access for ALL in ALS: A large-scale, inclusive, collaborative consortium to unlock the molecular and genetic mechanisms of amyotrophic lateral sclerosis.
James D Berry, Sabrina Paganoni, Matthew B Harms, Neil Shneider, Jinsy Andrews, Jeremy Shefner

Muscle Nerve· December 2024

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Automated speech analytics in ALS: higher sensitivity of digital articulatory precision over the ALSFRS-R.
Gabriela Stegmann, Chelsea Krantsevich, Julie Liss, Sherman Charles, Meredith Bartlett, Jeremy Shefner

Amyotroph Lateral Scler Frontotemporal Degener· November 2024

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Failures to Replicate: What Recent Negative Phase 3 Trials Have Taught Us about Amyotrophic Lateral Sclerosis Clinical Research.
Jeremy M Shefner, Merit E Cudkowicz

Ann Neurol· August 2024

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Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial.
Michael Benatar, Thomas Hansen, Dror Rom, Marie A Geist, Thomas Blaettler, Jeremy M Shefner

Lancet Neurol· July 2024

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The Miami Framework for ALS and related neurodegenerative disorders: an integrated view of phenotype and biology.
Michael Benatar, Joanne Wuu, Edward D Huey, Corey T McMillan, Ronald C Petersen, Jeremy Shefner

Nat Rev Neurol· June 2024

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Publisher Correction: The Miami Framework for ALS and related neurodegenerative disorders: an integrated view of phenotype and biology.
Michael Benatar, Joanne Wuu, Edward D Huey, Corey T McMillan, Ronald C Petersen, Jeremy Shefner

Nat Rev Neurol· June 2024

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The ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials.
Angela Genge, Jesse M Cedarbaum, Jeremy Shefner, Adriano Chio, Ammar Al-Chalabi

Amyotroph Lateral Scler Frontotemporal Degener· May 2024

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Relationship between quantitative strength and functional outcomes in the phase 2 FORTITUDE-ALS trial.
Jeremy M Shefner, Bill Jacobsen, Stuart Kupfer, Fady I Malik, Lisa Meng

Amyotroph Lateral Scler Frontotemporal Degener· February 2024

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Harmonized standard operating procedures for administering the ALS functional rating scale-revised.
Jeremy M Shefner, Tommy Bunte, Gale Kittle, Angela Genge, Leonard H van den Berg

Amyotroph Lateral Scler Frontotemporal Degener· February 2024

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Biomarker Qualification for Neurofilament Light Chain in Amyotrophic Lateral Sclerosis: Theory and Practice.
Michael Benatar, Lyle W Ostrow, Joseph W Lewcock, Frank Bennett, Jeremy Shefner

Ann Neurol· February 2024

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Skeletal muscle in amyotrophic lateral sclerosis.
Jeremy M Shefner, Antonio Musaro, Shyuan T Ngo, Christian Lunetta, Frederik J Steyn

Brain· November 2023

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A speech-based prognostic model for dysarthria progression in ALS.
Gabriela Stegmann, Sherman Charles, Julie Liss, Jeremy Shefner, Seward Rutkove

Amyotroph Lateral Scler Frontotemporal Degener· June 2023

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