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Neurology|July 1, 2016
International consensus guidance for management of myasthenia gravis: Executive summaryDonald B Sanders, Gil I Wolfe, Michael Benatar, et al.
Muscle & Nerve|April 25, 2020
Spinal muscular atrophy care in the COVID-19 pandemic eraAravindhan Veerapandiyan, Anne M Connolly, Richard S Finkel, et al.
Human Molecular Genetics|June 6, 2013
The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse modelsRocky G Gogliotti, Herminio Cardona, Jasbir Singh, et al.
BMC Neurology|November 15, 2013
Quantitative MRI analysis in children with multiple sclerosis: a multicenter feasibility pilot studyTanuja Chitnis, Charles R Guttmann, Alexander Zaitsev, et al.
Muscle & Nerve|June 22, 2020
The CINRG Becker Natural History Study: Baseline characteristicsPaula R Clemens, Gabriela Niizawa, Jia Feng, 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.
Eclinicalmedicine|May 21, 2026
The phenotypic spectrum and genetic determinants of severe spinal muscular atrophy in individuals with a single SMN2 copy: an international retrospective observational studyGianpaolo Cicala, Anna Capasso, Marianna Villa, et al.
Contemporary Clinical Trials Communications|August 11, 2018
Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!Amy Bartlett, Stephen J Kolb, Allison Kingsley, et al.
The Lancet. Neurology|August 16, 2025
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trialThomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
Pharmacological Research|September 17, 2018
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drugLaurie S Conklin, Jesse M Damsker, Eric P Hoffman, et al.
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