DYNE-251 clinical trials

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

This study is looking at a new medicine called DYNE-251 for boys aged 4-16 with Duchenne Muscular Dystrophy (DMD). It aims to see if the medicine is safe, well-tolerated, and helps the body make more dystrophin protein.