Duchenne Muscular Dystrophy research hub
4 indexed studies · 4 currently recruiting
- recruitingPHASE2
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
This study is testing a new medicine, NS-089/NCNP-02, for Duchenne muscular dystrophy (DMD) in boys aged 4 to 14. It's for those with a specific gene change (exon 44 skipping) and involves weekly IV infusions to see if the medicine is safe and helps their condition.
United States · Australia · Canada - 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.
France · Germany · Italy - recruiting
Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies
This study looks at how Duchenne (DMD) and Becker (BMD) muscular dystrophies affect thinking, feelings, and behaviour. It uses computer tasks and questionnaires to understand how the brain responds and to help create better ways to measure these effects in future research.
United Kingdom - 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