Affiliation: | 1. Center for Genetic Medicine, Children's National Health System, Washington, DC, USA;2. Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University, Binghamton, NY, USA;3. Center for Genetic Medicine, Children's National Health System, Washington, DC, USA Institute for Biomedical Sciences, The George Washington University, School of Medicine and Health Sciences, Washington, DC, USA;4. Center for Genetic Medicine, Children's National Health System, Washington, DC, USA Institute for Biomedical Sciences, The George Washington University, School of Medicine and Health Sciences, Washington, DC, USA Department of Genomics and Precision Medicine, The George Washington University, School of Medicine and Health Sciences, Washington, DC, USA Department of Pediatrics, The George Washington University, School of Medicine and Health Sciences, Washington, DC, USA;5. Center for Genetic Medicine, Children's National Health System, Washington, DC, USA Center for Translational Science, Children's National Health System, Washington, DC, USA;6. Department of Neurology, McColl-Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular/ALS Center, Carolinas Medical Center, Charlotte, NC, USA;7. Center for Genetic Medicine, Children's National Health System, Washington, DC, USA Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University, Binghamton, NY, USA |