GNT0004 (1x10e13 vg/kg) clinical trials

A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy

This study is testing a new gene therapy, GNT0004, for boys aged 6-10 with Duchenne Muscular Dystrophy. It aims to deliver a working 'mini-dystrophin' gene to muscles, hoping to slow down the disease. Researchers are looking at its safety and how well it works compared to a dummy treatment.