Deramiocel Can Safely Slow Progression in Advanced Duchenne Muscular Dystrophy

WEDNESDAY, Aug. 12, 2026 (HealthDay News) -- Deramiocel, a heart-derived cellular therapy consisting of human allogeneic cardiosphere-derived cells, can safely slow progression of advanced Duchenne muscular dystrophy (DMD), according to a study published online July 29 in The Lancet.Craig M. McDonald, M.D., from the University of California in Sacramento, and colleagues conducted a multicenter, randomized, double-blind, placebo-controlled study (HOPE-3) involving participants aged 10 years or older with DMD to examine the efficacy and safety of deramiocel. In outpatient settings, the investigational product was infused intravenously every three months.Overall, 106 participants were randomly assigned to deramiocel or placebo (54 and 52, respectively) and were included in the intention-to-treat population. The researchers found that the primary end point (total Performance of the Upper Limb 2.0 [PUL2.0]) percentage change from baseline showed significant improvement in the deramiocel group versus the placebo group. The least-squares mean percentage change at 12 months favored deramiocel by 4.55 percent for total PUL2.0. The safety profile was similar for deramiocel and placebo."The benefits we observed could mean important improvements in quality of life in people with advanced Duchenne," McDonald said in a statement.Several authors of The Lancet study disclosed ties to biopharmaceutical companies, including Capricor Therapeutics, which is developing deramiocel and funded the study.Abstract/Full Text.Sign up for our weekly HealthDay newsletter

Deramiocel Can Safely Slow Progression in Advanced Duchenne Muscular Dystrophy

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