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Annals of Clinical and Translational Neurology|August 20, 2025
Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History ComparisonsCraig M McDonald, Michela Guglieri, Dragana Vučinić, et al.
Human Mutation|October 6, 2011
Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD geneKevin M Flanigan, Diane M Dunn, Andrew von Niederhausern, et al.
Steroids|November 20, 2025
Exposure-response of serum biomarkers to vamorolone, a dissociative corticosteroidal anti-inflammatory drug, in 4- to <7-year childrenSwati Mummidivarpu, Utkarsh J Dang, Michael Ziemba, et al.
Muscle & Nerve|January 25, 2019
Revised upper limb module for spinal muscular atrophy: 12 month changesMaria Carmela Pera, Giorgia Coratti, Elena S Mazzone, et al.
Brain : a Journal of Neurology|August 28, 2015
Genotype-phenotype characteristics and baseline natural history of heritable neuropathies caused by mutations in the MPZ geneOranee Sanmaneechai, Shawna Feely, Steven S Scherer, et al.
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics|February 24, 2021
Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating StudyStacy A Rudnicki, Jinsy A Andrews, Tina Duong, et al.
Plos One|April 9, 2013
SMA-MAP: a plasma protein panel for spinal muscular atrophyDione T Kobayashi, Jing Shi, Laurie Stephen, et al.
The Lancet. Neurology|March 20, 2021
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trialJohn W Day, Richard S Finkel, Claudia A Chiriboga, et al.
Neurology|September 8, 2021
Association Between Body Mass Index and Disability in Children With Charcot-Marie-Tooth DiseaseGabrielle A Donlevy, Sarah P Garnett, Kayla M D Cornett, et al.
Brain : a Journal of Neurology|October 22, 2021
Preventing amyotrophic lateral sclerosis: insights from pre-symptomatic neurodegenerative diseasesMichael Benatar, Joanne Wuu, Caroline McHutchison, et al.
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