Accessibility StatementSkip Navigation -- Unprecedented improvement compared to baseline and natural history in multiple functional measures including Time to Rise from Floor (TTR), 4-Stair Climb (4SC), Performance of Upper Limb (PUL) and 10- Meter Walk/Run Test (10mWRT) at approximately one year -- -- Unprecedented rapid reduction in creatine kinase (CK) to near normal levels maintained over 16 months of follow-up and 25% increase of normal in dystrophin production, reflecting sustained muscle fiber protec ...