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Annals of Neurology|November 18, 2017
Natural history of infantile-onset spinal muscular atrophyStephen J Kolb, Christopher S Coffey, Jon W Yankey, et al.Annals of Clinical and Translational Neurology|February 23, 2016
Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker studyStephen J Kolb, Christopher S Coffey, Jon W Yankey, et al.Neurology|February 9, 2024
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled TrialUtkarsh J Dang, Jesse M Damsker, Michela Guglieri, et al.Acta Neuropathologica|April 29, 2023
AMFR dysfunction causes autosomal recessive spastic paraplegia in human that is amenable to statin treatment in a preclinical modelRuizhi Deng, Eva Medico-Salsench, Anita Nikoncuk, et al.Pageof 4