Mendell JR, Sahenk Z, Lehman K, et al. - In this open-label, phase 1/2a nonrandomized controlled trial, researchers identified the 1-year safety and tolerability of intravenous rAAVrh74.MHCK7.micro-dystrophin in patients with Duchenne muscular dystrophy (DMD). This trial was carried out at the Nationwide Children’s Hospital in Columbus, Ohio. The sample consisted of 4 young patients (mean [SD] age at enrollment, 4.8 [1.0] years) with DMD. This trial demonstrated that rAAVrh74.MHCK7.micro-dystrophin to be well tolerated and have least adverse events; the safe delivery of micro-dystrophin transgene; the robust expression and correct localization of micro-dystrophin protein; and improvements in creatine kinase levels and NSAA scores. Such results indicate that rAAVrh74.MHCK7.micro-dystrophin may provide functional improvement that is greater than that observed under a standard of care.