Duchenne Muscular Dystrophy research hub
4 indexed studies · 2 currently recruiting
- recruitingPHASE2
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
This study is testing a new medicine, SAT-3247, for boys aged 7-9 with Duchenne muscular dystrophy. We want to find the best dose, see if it's safe, and check if it helps. Some boys will get the real medicine, others a dummy medicine.
United States · Australia · Belgium - 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.
Belgium · France · Germany - active not recruiting
Defining Endpoints in Becker Muscular Dystrophy
This study looks at Becker muscular dystrophy over two years. It aims to understand how the condition changes over time in 50 people. This will help doctors plan better treatments in the future. It doesn't involve new medicines.
United States · New Zealand · United Kingdom - active not recruitingPHASE2
Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
This study is checking a new medicine called AOC 1020 for people with Facioscapulohumeral Muscular Dystrophy (FSHD). It's for people who were in an earlier study of this medicine. The aim is to see if it's safe and helps over a longer time.
United States · Canada · United Kingdom