Sarepta Shifts Focus to siRNA Platform, Lays Off 500 After Elevidys Receives Black Box Warning

Sarepta Therapeutics; siRNA platform; Elevidys; Duchenne muscular dystrophy; layoffs; black box warning; pipeline shift; acute liver injury; gene therapy; restructuring

FDA Rejects Capricor’s Cell Therapy for Duchenne Muscular Dystrophy

FDA rejection; Capricor Therapeutics; deramiocel; Duchenne muscular dystrophy; DMD; cell therapy; cardiomyopathy; clinical data; Phase 3 HOPE-3 trial; Complete Response Letter

FDA Investigates Patient Deaths Linked to Sarepta’s Elevidys Gene Therapy for Duchenne Muscular Dystrophy

FDA; Sarepta; Elevidys; Duchenne muscular dystrophy; gene therapy; patient deaths; acute liver failure; liver toxicity; regulatory investigation; non-ambulatory patients

Second Patient Dies After Receiving Sarepta’s Elevidys Gene Therapy for DMD

Duchenne muscular dystrophy; gene therapy; Sarepta Therapeutics; Elevidys; acute liver failure; AAV-based gene therapy; patient death; non-ambulatory patients; FDA; risk mitigation

Sarepta Halts Duchenne Gene Therapy in Some Patients After Second Death

Sarepta Therapeutics; gene therapy; Duchenne muscular dystrophy; Elevidys; acute liver failure; non-ambulatory patients; FDA; clinical trial pause; immunosuppression; patient safety

Wave Life Sciences’ WVE-N531 Shows Promise in Duchenne Muscular Dystrophy Treatment

Duchenne muscular dystrophy (DMD), WVE-N531, exon 53 skipping, Phase II trial, dystrophin expression, muscle health improvement, FDA approval