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Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial.

PubMed / Lancet · 2026-07-29 · By McDonald CM, Villa C, Soslow JH, et al. · Lancet (London, England)

The HOPE-3 phase 3 randomized controlled trial evaluated deramiocel, a heart-derived cellular therapy, in patients with advanced Duchenne muscular dystrophy (DMD). Published in The Lancet, the double-blind, placebo-controlled study examined the cardiac and functional effects of this investigational treatment in a population with significant disease progression. The findings contribute to the evidence base for novel disease-modifying approaches in DMD, a condition that often involves physical therapy as part of multidisciplinary management.

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