Three boys, three forms of muscular dystrophy — and one medicine reaching all their muscles

For the first time in any human disease, a new kind of RNA medicine has switched a person's own gene on — not off. Three boys, ages 4 to 15, living with Duchenne muscular dystrophy received RAG-18 as a monthly drip. Biopsies of their leg muscles showed their own utrophin protein — a natural stand-in for the dystrophin their bodies cannot make — rising three to five times over. What moved the researchers: each boy carries a different form of the disease, yet all three responded in the same direction, hinting at a treatment that could one day help every child with Duchenne, whatever their mutation. No serious side effects were reported. The results were presented today in Hiroshima, Japan, at the World Muscle Society congress.