Innovative therapy for Classic Galactosemia - Tale of two HTS

► Unsatisfactory outcome in Classic Galactosemia calls for a more effective therapy. ► Human galactokinase (GALK) provides a validated target for intervention. ► Experimental and computational HTS for GALK inhibitors were performed. ► Characterization and optimization of selected positives identified promising leads. ► GALK inhibitors can be developed into novel antimicrobials/anti-cancer therapies.