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 ...