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Muscle & Nerve|September 18, 2009
An analysis of disease severity based on SMN2 copy number in adults with spinal muscular atrophyBakri Elsheikh, Thomas Prior, Xiaoli Zhang, et al.Neurology|August 28, 2016
Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophyRobert C Griggs, J Phillip Miller, Cheryl R Greenberg, et al.Annals of Neurology|June 3, 2010
Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophyVinod Malik, Louise R Rodino-Klapac, Laurence Viollet, et al.Biorxiv : the Preprint Server for Biology|July 3, 2026
Metabolomic Network Analysis Reveals Reorganization of Lipid and Steroid Programs Linked to Right Ventricular-Pulmonary Vascular Function in Pulmonary HypertensionIvor W Clinton, Julie C Coursen, Darin T Rosen, et al.Journal of Neuropathology and Experimental Neurology|October 6, 2006
Limb-girdle muscular dystrophy in the United StatesSteven A Moore, Christopher J Shilling, Steven Westra, et al.The Lancet. Neurology|February 21, 2023
Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trialCharles A Thornton, Richard Thomas Moxley, Katy Eichinger, et al.Pageof 2