Gene therapy fails in DMD


United States

A gene therapy being investigated in a Phase 3 trial of boys with Duchenne muscular dystrophy (DMD) has failed to reach its primary endpoint of improvement in motor function, the developer Pfizer Inc announced on 12 June. The therapy, fordadistrogene movaparvovec, missed the primary endpoint at one year as well as key secondary endpoints compared with a placebo. The secondary endpoints included an improvement in velocity in a 10 metre run or walk. Participants were boys between the ages of four and seven who were on a daily regimen of glucocorticoids.