- recruitingPHASE3
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
This study looks at a new medicine called AOC 1044 (or delpacibart zotadirsen) for boys with Duchenne Muscular Dystrophy (DMD) who have a specific genetic change (exon 44 skipping). It aims to see if the medicine is safe and helps improve the condition, compared to a dummy medicine.
Australia · Belgium · France - recruitingPHASE2, PHASE3
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.
Belgium · France · Spain