Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial
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The authors aimed to evaluate the efficacy and safety of deramiocel, a heart-derived cellular therapy, in patients with advanced Duchenne muscular dystrophy (DMD). In a phase 3, randomized, double-blind, placebo-controlled trial, they found that deramiocel significantly improved skeletal muscle function compared to placebo, with a safety profile similar to that of the control group. These results suggest that deramiocel may effectively slow disease progression in advanced DMD.
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