Omar Khwaja, an accomplished pediatric neurologist, researcher, and drug developer, is determined to help patients with high-unmet medical needs. With extensive knowledge in critical care, fetal-neonatal neurology, neurogenetics, and developmental neurobiology, Khwaja has spearheaded numerous clinical-translational research initiatives. Khwaja's expertise lies mainly in diseases related to the developing brain, rare diseases, and neurodevelopmental disorders, with a particular focus on molecular genetic and neuroscientific approaches to drug development. Khwaja is the former Chief Medical Officer at Voyager Therapeutics and SMA Foundation and has served in significant leadership roles in Roche Pharmaceuticals Research and Early Development.