A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
- EligibilityCriteria: Inclusion Criteria:  Age 4 to 10 Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping Able to walk Not requiring assistance from a ventilator to breathe Currently on consistent doses of steroid treatment for the last 12 weeks  Exclusion Criteria:  The participant will have some initial clinical labs and studies to assess baseline level of heart and lung function. Treatment with an exon skipping therapy within 12 weeks prior to the first visit. Any history of treatment with gene therapy - HealthyVolunteers: No - Gender: Male - MinimumAge: 4 Years - MaximumAge: 10 Years - StdAgeList: Child