A physician scientist is making strides in understanding the molecular origins of fatty liver disease, a leading cause of liver failure in the United States. An Indiana University School of Medicine ...
One year ago this week, Baby KJ received his very first infusion of a gene therapy made specifically for him. 1 He had a unique mutation that led to carbamoyl-phosphate synthetase 1 (CPS1) deficiency, ...
Developing high-quality, safe, and effective drugs is a complex process that requires varied scientific skills and stringent regulatory assessments. Drug development is a process that spans many years ...