authors Hughes, Meghan C Ramos, Sofhia V Brahmbhatt, Aditya Turnbull, Patrick C Polidovitch, Nazari N Garibotti, Madison C Schlattner, Uwe Hawke, Thomas Simpson, Jeremy A Backx, Peter H Perry, Christopher GR
keywords 2.1 Biological and endogenous factors 31 Biological Sciences 3101 Biochemistry and Cell Biology 32 Biomedical and Clinical Sciences 3208 Medical Physiology 5.1 Pharmaceuticals Cardiovascular Duchenne/ Becker Muscular Dystrophy Muscular Dystrophy Orphan Drug Pediatric Rare Diseases