Muscular Dystrophy, Duchenne
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
Taiho’s Investigational DMD Therapy Misses Primary Endpoint in Phase 3 Trial
Taiho; Duchenne muscular dystrophy; DMD; pizuglanstat; TAS-205; Phase 3 trial; clinical trial failure; REACH-DMD study; Otsuka Holdings
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
Regenxbio Reports Consistent One-Year Benefit for DMD Gene Therapy RGX-202
Regenxbio; DMD; Duchenne muscular dystrophy; RGX-202; gene therapy; AFFINITY DUCHENNE trial; clinical trial; microdystrophin; biomarker; phase I/II; functional data
Entrada tightens focus on Duchenne, axes 20% of staff
Duchenne muscular dystrophy (DMD), workforce reduction, ENTR-601-44, ENTR-601-45, FDA clinical hold, exon skipping therapy
Biotech Restructuring and European Regulatory Setbacks
LifeMine Therapeutics, layoffs, PTC Therapeutics, Translarna, Duchenne muscular dystrophy, European Commission, regulatory decision
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