Highlights
The Phase 3 CIFFREO trial evaluated fordadistrogene movaparvovec, an investigational AAV9-based mini-dystrophin gene therapy, in 122 ambulatory boys with Duchenne muscular dystrophy (DMD).
The study failed to meet its primary endpoint, with no statistically significant difference in the North Star Ambulatory Assessment (NSAA) total score change from baseline to week 52 between the treatment and placebo groups (p=0.91).
