Muscular Dystrophy, Duchenne
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
Sarepta Reports Death of DMD Patient Following Elevidys Gene Therapy Treatment
Sarepta, Elevidys, gene therapy, Duchenne muscular dystrophy, patient death, acute liver failure
Avidity’s Del-zota Shows Promise in Duchenne Muscular Dystrophy Treatment
Del-zota, Duchenne muscular dystrophy (DMD), RNA therapeutics, exon 44 skipping, dystrophin production, creatine kinase reduction, safety profile
Entrada given go-ahead to test DMD therapy after two-year hold
Entrada, Muscular Dystrophy, Duchenne, DMD, United States Food and Drug Administration, Adult, Exons
FDA Lifts Clinical Hold on Entrada’s Duchenne Muscular Dystrophy Drug ENTR-601-44 After Two Years
Duchenne muscular dystrophy, ENTR-601-44, Entrada Therapeutics, FDA, clinical hold, exon 44 skipping, ELEVATE-44-102, Phase 1b trial