Unable to walk (finding)

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

1 month ago talkbio0Tagged ELEVIDYS, Evaluation, gene therapy, Liver, Liver Failure, Acute, Muscular Dystrophy, Duchenne, patient deaths, regulatory, Toxicity aspects, Unable to walk (finding), United States Food and Drug Administration

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

1 month ago talkbio0Tagged AAV-based gene therapy, ELEVIDYS, gene therapy, Liver Failure, Acute, Muscular Dystrophy, Duchenne, Patient Death, risk mitigation, Unable to walk (finding), United States Food and Drug Administration

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

1 month ago talkbio0Tagged Clinical Trials, ELEVIDYS, gene therapy, Liver Failure, Acute, Muscular Dystrophy, Duchenne, patient safety, Therapeutic immunosuppression, Unable to walk (finding), United States Food and Drug Administration

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

Sarepta Therapeutics: Next Steps After Full FDA Approval for Duchenne Muscular Dystrophy Gene Therapy

1 year ago talkbio0Tagged Ambulatory - qualifier value, Approved, Duchenne, ELEVIDYS, gene therapy, Genetic Medicine, Microdystrophin, pipeline development, Unable to walk (finding), United States Food and Drug Administration

Sarepta Therapeutics, Duchenne muscular dystrophy, gene therapy, FDA approval, Elevidys, microdystrophin, ambulatory patients, non-ambulatory patients, pipeline development, rare diseases, genetic medicine.

FDA Expands Approval of Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy to Include Older Boys and Non-Ambulatory Patients

1 year ago talkbio0Tagged Ambulatory - qualifier value, Approved, Duchenne, ELEVIDYS, gene therapy, Muscular Dystrophy, Unable to walk (finding), United States Food and Drug Administration

FDA, Sarepta, Duchenne Muscular Dystrophy, Gene Therapy, Elevidys, Ambulatory, Non-Ambulatory, Expanded Approval