Randomized placebo controlled trial· 2017
Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: results of a randomized, placebo-controlled clinical trial
Campbell C, McMillan HJ, Mah JK, et al.
Muscle and Nerve
- PubMed
- 27462804
- Evidence level
- moderate
- Verification
- verified
Summary
Ascending-dose randomized trial of ACE-031 in ambulatory boys with Duchenne muscular dystrophy, primarily evaluating safety.
Limitations
Premature termination, small sample, short duration and exploratory functional outcomes.