Capricor Therapeutics’ donor-cell bloodstream therapy deramiocel produced statistically significant slowing of upper-limb functional decline in boys and young men with advanced Duchenne muscular dystrophy in the HOPE-3 phase 3 trial, published in The Lancet. The randomized study enrolled 106 patients ages 10 to 22 across 20 U.S. sites and delivered deramiocel via IV drip every three months for one year versus placebo. After 12 months, deramiocel-treated participants declined more slowly in arm movement: overall arm function declined about 54% more slowly than placebo, with elbow movement about 65% more slowly. A clearer differentiation of cardiac outcomes emerged in subgroups with baseline heart disease and analyzable cardiac scans, where heart function was better preserved versus placebo. The therapy also appeared to reduce markers consistent with heart scarring expansion in a smaller set of patients with before-and-after scans. Across the trial, no deaths were reported and safety was generally favorable, though allergic-type reactions were more common in the deramiocel arm (42% vs 15%), with most events mild or moderate. Regulatory review pressure remains, as the FDA has already posted a critical analysis challenging how effectively deramiocel separated from placebo on key endpoints, according to public review materials discussed in parallel coverage ahead of an FDA advisory committee.