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Neurology|January 4, 2020
Safety and efficacy of omaveloxolone in patients with mitochondrial myopathy: MOTOR trialKaren L Madsen, Astrid E Buch, Bruce H Cohen, et al.
Muscle & Nerve|May 19, 2022
Quantitative muscle analysis in facioscapulohumeral muscular dystrophy using whole-body fat-referenced MRI: Protocol development, multicenter feasibility, and repeatabilityPer Widholm, André Ahlgren, Markus Karlsson, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 30, 2025
Complement activation in a phase Ib study of fordadistrogene movaparvovec for Duchenne muscular dystrophyBarry J Byrne, Russell J Butterfield, Perry B Shieh, et al.
Neuromuscular Disorders : NMD|March 8, 2021
Disease-modifying effects of edasalonexent, an NF-κB inhibitor, in young boys with Duchenne muscular dystrophy: Results of the MoveDMD phase 2 and open label extension trialRichard S Finkel, Erika Finanger, Krista Vandenborne, et al.
Neurology|June 24, 2022
Quantitative Muscle Analysis in FSHD Using Whole-Body Fat-Referenced MRI: Composite Scores for Longitudinal and Cross-sectional AnalysisMichelle L Mellion, Per Widholm, Markus Karlsson, et al.
Neurology|August 27, 2013
Recessive truncating titin gene, TTN, mutations presenting as centronuclear myopathyOzge Ceyhan-Birsoy, Pankaj B Agrawal, Carlos Hidalgo, et al.
Journal of Neuromuscular Diseases|March 13, 2023
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)Richard S Finkel, Basil T Darras, Jerry R Mendell, et al.
Journal of Neuromuscular Diseases|January 22, 2024
Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE RegistryLaurent Servais, John W Day, Darryl C De Vivo, et al.
Neuromuscular Disorders : NMD|February 20, 2025
Strength and functional correlates of reachable workspace in facioscapulohumeral muscular dystrophyLeo H Wang, Maya N Hatch, Michael P McDermott, et al.
Human Gene Therapy|February 13, 2026
Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and ManagementPerry B Shieh, Crystal Proud, Tamir Diamond, et al.
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