We recently rated a one-time gene therapy for Duchenne muscular dystrophy in young boys.

The Phase 3 trial was well designed. We looked at the primary endpoint. It was not met. Overall motor function did not improve significantly.

We looked at secondary endpoints. Some specific motor tests showed improvement.

We looked at quality-of-life data. There was none.

We looked at the cost. $3.2 million. The cost per outcome gained is projected well above standard thresholds.

The secondary data suggested something. The primary data did not confirm it.

Read the full MARA Rating here: https://mararating.com/report/elevidys-delandistrogene-moxeparvovec-for-duchenne-muscular-dystrophy-dmd-as-of-january-2026/