Why carry out this study?

Duchenne muscular dystrophy (DMD) is a genetic muscle disorder that manifests during early childhood and is ultimately fatal

Approved therapies for DMD are only available for specific populations of patients and an unmet need remains for those patients who are not able to receive available treatments

The aim of the studies was to evaluate the safety and efficacy of the anti-myostatin taldefgrobep alfa as a potential treatment for patients with DMD

What was learned from the study?

The phase 2/3 trial of taldefgrobep alfa in boys with DMD did not meet the primary endpoint of a functional change assessed using the North Star Ambulatory Assessment and the trial was subsequently terminated

Although taldefgrobep alfa did not result in functional improvements in boys with DMD, there were no safety concerns identified from the treatment