Four clinical trials based on micro-dystrophin gene therapy are currently underway10, 11, 12 (NCT03368742 Solid Biosciences; NCT03375164, NCT03769116, NCT04626674, and NCT05096221 Sarepta Therapeutics; NCT03362502, NCT04281485, and NCT05429372 Pfizer, EudraCT Number 2020-002093-27 Genethon; and NCT05693142 Regenxbio), and very recently Sarepta Therapeutics announced US Food and Drug Administration approval of ELEVIDYS, the first gene therapy to treat DMD. Here, DMD is linked to Duchenne muscular dystrophy.