Recently published data on four young DMD patients followed-up for 12 months after a rAAVrh74.MHCK7.micro-dystrophin gene transfer showed that this treatment was well-tolerated, and was associated with robust micro-dystrophin expression, reduced serum CK levels, and functional improvement as measured by the North Star Ambulatory Assessment (68). This evidence concerns the gene DMD and Duchenne muscular dystrophy.