Taiho Pharmaceutical reported the failure of its phase III trial for pizuglanstat, an experimental therapy for Duchenne muscular dystrophy (DMD). The study did not meet its primary endpoint measuring motor function improvement in ambulatory patients. This setback adds to the ongoing challenges in developing effective therapies for DMD. Despite promising preclinical models, translating efficacy into clinical benefit remains difficult for treatments targeting this rare and severe genetic muscle-wasting disease.