www.fdanews.com/articles/102610-muscular-dystrophy-study-results-published
Muscular Dystrophy Study Results Published
December 27, 2007
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.