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The Journal of Biological Chemistry|March 29, 2012
Novel mutation in spectrin-like repeat 1 of dystrophin central domain causes protein misfolding and mild Becker muscular dystrophyGyula Acsadi, Steven A Moore, Angélique Chéron, et al.Human Gene Therapy|June 18, 2002
Increased survival and function of SOD1 mice after glial cell-derived neurotrophic factor gene therapyGyula Acsadi, Roumen A Anguelov, Huibin Yang, 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.Clinical Biomechanics (Bristol, Avon)|March 20, 2012
Symmetry of foot alignment and ankle flexibility in paediatric Charcot-Marie-Tooth diseaseJoshua Burns, Robert Ouvrier, Tim Estilow, et al.Human Mutation|July 26, 2011
Functional consequences and structural interpretation of mutations of human choline acetyltransferaseXin-Ming Shen, Thomas O Crawford, Joan Brengman, et al.Annals of Neurology|April 24, 2012
Validation of the Charcot-Marie-Tooth disease pediatric scale as an outcome measure of disabilityJoshua Burns, Robert Ouvrier, Tim Estilow, et al.Journal of Child Neurology|September 1, 2007
Perspectives on clinical trials in spinal muscular atrophyKathryn J Swoboda, John T Kissel, Thomas O Crawford, et al.Muscle & Nerve|August 26, 2010
Compound muscle action potential and motor function in children with spinal muscular atrophyAga Lewelt, Kristin J Krosschell, Charles Scott, et al.Plos One|May 15, 2009
Phase II open label study of valproic acid in spinal muscular atrophyKathryn J Swoboda, Charles B Scott, Sandra P Reyna, et al.Plos One|July 15, 2011
SMA CARNIVAL TRIAL PART II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophyJohn T Kissel, Charles B Scott, Sandra P Reyna, et al.Pageof 5