Signal Cedars-Sinai Phase 3 trial shows deramiocel cell therapy preserves cardiac function in Duchenne muscular dystrophy

Summary

Results of a Phase 3 clinical trial called HOPE-3, testing the cell therapy deramiocel for Duchenne muscular dystrophy, were published in The Lancet. The trial was led by researchers at Cedars-Sinai. Compared with placebo, the therapy was shown to slow loss of upper-limb muscle function and to protect against decline in cardiac function among pediatric and adolescent patients. The therapy is being characterized as the first treatment proven to preserve cardiac function in Duchenne muscular dystrophy patients, a leading cause of mortality in the disease. Duchenne muscular dystrophy is a severe, degenerative genetic muscle-wasting disease, and the trial represents a large randomized study supporting a cell-therapy approach for a rare pediatric disease with significant unmet need.