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Pediatric Neurology|July 7, 2019
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement TherapyLinda P Lowes, Lindsay N Alfano, W David Arnold, 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.Pediatric Neurology|May 21, 2014
One year outcome of boys with Duchenne muscular dystrophy using the Bayley-III scales of infant and toddler developmentAnne M Connolly, Julaine M Florence, Mary M Cradock, et al.The Journal of Neuroscience : the Official Journal of the Society for Neuroscience|April 15, 2016
The Adaptor Protein CD2AP Is a Coordinator of Neurotrophin Signaling-Mediated Axon Arbor PlasticityBenjamin J Harrison, Gayathri Venkat, James L Lamb, et al.Human Gene Therapy|July 15, 2017
Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for DysferlinopathyRachael A Potter, Danielle A Griffin, Patricia C Sondergaard, et al.Journal of Neuromuscular Diseases|April 12, 2024
Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular DystrophyCraig M Zaidman, Natalie L Goedeker, Amal A Aqul, et al.Human Mutation|September 18, 2010
Novel LMNA mutations in patients with Emery-Dreifuss muscular dystrophy and functional characterization of four LMNA mutationsJuergen Scharner, Charlotte A Brown, Matthew Bower, et al.Nature Medicine|January 4, 2024
Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial resultsJerry R Mendell, Eric R Pozsgai, Sarah Lewis, et al.Neurology|November 10, 2004
Correlating phenotype and genotype in the periodic paralysesT M Miller, M R Dias da Silva, H A Miller, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|August 4, 2011
Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disordersAdam K Bevan, Sandra Duque, Kevin D Foust, et al.Pageof 89