- Active not recruitingPHASE1, PHASE2
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
This study is looking for boys aged 4-10 with Duchenne muscular dystrophy (DMD) with a specific genetic change. It's testing a new medicine called BMN 351 to see how safe it is and how well the body handles it. They want to find the right dose for future studies.
For: Duchenne Muscular DystrophyItaly · Netherlands · Spain - Enrolling by invitationPHASE2
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This study looks at the long-term effects of a medicine called BMN 351 for boys and young men with Duchenne Muscular Dystrophy. It's for those who have already started taking BMN 351 in an earlier study and will continue to check its safety and how well it helps with physical movement.
For: Duchenne Muscular Dystrophy (DMD)Italy · Netherlands · Spain