DMD is the most common dystrophy,318 and the FDA recently granted approval for an rAAV-based gene therapy.319 DMD is a rare X-linked and progressive disease with a prevalence of ~15.9–19.5/100,000,320 which primarily affects males, although rare cases in females have been reported.321 It is caused by mutations in the dystrophin gene resulting in absent or dysfunctional dystrophin protein. This evidence concerns the gene DMD and Duchenne muscular dystrophy.