A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy. A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tole ...
Duchenne Muscular Dystrophy
Drug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Group 4;Drug: Regimen Selection Phase Group 1 (COMPLETED);Drug: DosingExtensionDrug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Gr ...