Overview

A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

Status:
Terminated
Trial end date:
2016-08-03
Target enrollment:
Participant gender:
Summary
The purpose of the study is to see whether BMN053 is safe and effective to use as medication for Duchenne muscular dystrophy (DMD) patients with a mutation around location 53 in the DNA for the dystrophin protein.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
BioMarin Pharmaceutical