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JAMA Neurology|February 9, 2026
Long-Term Tofersen in SOD1 Amyotrophic Lateral SclerosisTimothy M Miller, Merit E Cudkowicz, Pamela J Shaw, et al.
Journal of Parkinson'S Disease|December 24, 2019
The Quebec Parkinson Network: A Researcher-Patient Matching Platform and Multimodal BiorepositoryZiv Gan-Or, Trisha Rao, Etienne Leveille, et al.
The Lancet. Neurology|July 26, 2024
Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose studyLeonard H van den Berg, Jeffrey D Rothstein, Pamela J Shaw, et al.
The New England Journal of Medicine|July 9, 2020
Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALSTimothy Miller, Merit Cudkowicz, Pamela J Shaw, et al.
Muscle & Nerve|April 16, 2022
Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophyJeffrey M Statland, Craig Campbell, Urvi Desai, et al.
Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration|July 6, 2025
Toward therapeutic trials in primary lateral sclerosisErica Scirocco, Matti D Allen, Elisa Giacomelli, et al.
Journal of Neuromuscular Diseases|February 6, 2026
A randomized, double-blind, placebo-controlled study of losmapimod in patients with facioscapulohumeral muscular dystrophy: Results of the REACH studyNicol C Voermans, Jeffrey M Statland, Lawrence J Hayward, et al.
JAMA Neurology|March 24, 2025
Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical TrialJeremy M Shefner, Merit E Cudkowicz, Angela Genge, et al.
Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration|June 12, 2025
Hospitalizations as an outcome measure in COURAGE-ALSStacy A Rudnicki, Ammar Al-Chalabi, Jinsy A Andrews, et al.
Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration|May 31, 2023
COURAGE-ALS: a randomized, double-blind phase 3 study designed to improve participant experience and increase the probability of successJeremy M Shefner, Ammar Al-Chalabi, Jinsy A Andrews, et al.
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