Leiden University Medical Center (LUMC) and Dutch drugmaker Prosensa’s RNA-based therapeutic PRO051 restored dystrophin expression in patients with Duchenne Muscular Dystrophy.

The study included four patients aged 10–13 and is the first with an RNA-based therapeutic agent. The study’s results were published in The New England Journal of Medicine.

Prosensa said it is preparing for a Phase I/II study to explore the effects and safety of PRO051 after repeated systemic injections.