I always pay close attention to DMD research. The FDA granted Orphan Drug Designation to GEn-1123, an investigational therapy. This special status is for diseases affecting fewer than 200,000 people, and it helps speed up development.
The U.S. Food and Drug Administration (FDA) has awarded both Orphan Drug and Rare Pediatric Disease Designations to GEn-1123, an investigational oral therapy developed by GEn1E Lifesciences for Duchen
