Capricor Therapeutics experienced a major setback after the FDA rejected its cell therapy, Deramiocel, for treating heart disease in Duchenne muscular dystrophy (DMD) patients. The decision led to a 30% drop in Capricor’s stock and highlighted the need for more efficacy data. Deramiocel, which uses cardiac-derived stem cells, aims to regenerate heart tissue and slow cardiomyopathy progression in DMD. Capricor plans to resubmit its application with additional clinical data lat

Sarepta Therapeutics has paused its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys, after a second patient death due to acute liver failure. The pause affects ongoing trials and therapy shipments, raising safety concerns—especially for non-ambulatory patients. Approved under the FDA’s accelerated pathway, Elevidys aimed to slow DMD progression, but recent events have sparked debate over gene therapy risks, regulatory oversight, and treatment decisions for affected f