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Neurology|March 3, 2019
Myasthenic congenital myopathy from recessive mutations at a single residue in Na<sub>V</sub>1.4Nathaniel Elia, Johanna Palmio, Marisol Sampedro Castañeda, et al.Journal of Comparative Effectiveness Research|October 25, 2021
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophyPerry B Shieh, Gary Elfring, Panayiota Trifillis, et al.Neuromuscular Disorders : NMD|November 2, 2019
Large in-frame 5' deletions in DMD associated with mild Duchenne muscular dystrophy: Two case reports and a review of the literatureElizabeth M Gibbs, Florian Barthélémy, Emilie D Douine, et al.Journal of Comparative Effectiveness Research|February 7, 2023
Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional statusCraig M McDonald, Jessica R Marden, Perry B Shieh, et al.Muscle & Nerve|January 27, 2021
Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE studyGyula Acsadi, Thomas O Crawford, Wolfgang Müller-Felber, et al.Journal of Neuromuscular Diseases|November 11, 2025
Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registryYasemin Erbaş, Laurent Servais, Perry B Shieh, et al.Muscle & Nerve|October 11, 2019
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trialsCraig M McDonald, Gautam Sajeev, Zhiwen Yao, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|June 30, 2025
Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchenne muscular dystrophy: Initial observations from a phase 1b trialSarah P Sherlock, Daniel I Levy, Avery McIntosh, et al.Muscle & Nerve|November 11, 2017
Review of the Diagnosis and Treatment of Periodic ParalysisJeffrey M Statland, Bertrand Fontaine, Michael G Hanna, et al.Nature Medicine|June 27, 2025
AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trialRussell J Butterfield, Perry B Shieh, Huihua Li, et al.Pageof 9