Almost every recent advance in Duchenne muscular dystrophy has aimed at the same target: the faulty dystrophin gene. Exon-skipping drugs and gene therapy try to restore some version of the missing protein, and they work only for patients whose mutation matches.
A Phase 3 trial published in The Lancet took a different approach entirely. It left the mutation alone and tried to slow the damage instead, in boys and young men whose disease was already advanced.
