[Articles] Safety and efficacy of fordadistrogene movaparvovec in ambulatory participants with Duchenne muscular dystrophy (CIFFREO): a phase 3, double-blind, randomised, placebo-controlled study

The study did not meet its primary efficacy endpoint. Based on the efficacy and safety data from this phase 3 study, the benefit–risk profile of fordadistrogene movaparvovec was determined to be negative. Thus, the study sponsor has discontinued any further clinical development of this investigational gene therapy agent.