DMD gene therapy GNT0004 set to enter Phase 3 trial in Europe, US
GNT0004, an experimental gene therapy for Duchenne muscular dystrophy (DMD), appears to be working as intended in the initial parts of a multiphase clinical trial, with benefits including stable or improved motor function. That’s according to data presented by Genethon, the therapy’s developer, at the ASGCT Breakthroughs in…