AI Summary
Duchenne muscular dystrophy is a severe disorder affecting male babies. Gene therapy has been conditionally approved for boys aged 4-5, but more research is needed regarding its effectiveness and when to administer it.
Duchenne muscular dystrophy is a severe neuromuscular disorder that affects about one in 5000 male newborn babies.1 In June, 2023, the US Food and Drug Administration conditionally approved gene therapy with adeno-associated virus microdystrophin for boys aged 4–5 years; the phase 3 trial is ongoing.2 Despite this major milestone, treatments for boys with Duchenne muscular dystrophy are urgently needed. Furthermore, the magnitude and duration of effect of gene therapy, and the optimal age at which to deliver it, are uncertain.