ELEVIDYS – Page 2

Sarepta Reports Third Patient Death in Gene Therapy Studies for Limb-Girdle Muscular Dystrophy

1 week ago talkbio0Tagged AAVS1 gene, ELEVIDYS, gene therapy, Liver Failure, Acute, Muscular Dystrophies, Limb-Girdle, Patient Death, Safety, Study on Hold, United States Food and Drug Administration, vector

Sarepta Therapeutics; limb-girdle muscular dystrophy; SRP-9004; gene therapy; patient death; acute liver failure; FDA clinical hold; AAV vector; Elevidys; safety concerns

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

1 week ago talkbio0Tagged acute liver injury, black box, Cautionary Warning, ELEVIDYS, gene therapy, layoffs, Muscular Dystrophy, Duchenne, pipeline shift, restructuring, RNA, Small Interfering

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

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 Reports Death of DMD Patient Following Elevidys Gene Therapy Treatment

4 months ago talkbio0Tagged acute, ELEVIDYS, gene therapy, Liver Failure, Muscular Dystrophy, Duchenne

Sarepta, Elevidys, gene therapy, Duchenne muscular dystrophy, patient death, acute liver failure

Sarepta Therapeutics Reports Encouraging Two-Year Data for ELEVIDYS in Duchenne Muscular Dystrophy, Projecting $1 Billion in Sales

6 months ago talkbio0Tagged Clinical Trials, DMD, Duchenne, ELEVIDYS, gene therapy, Muscular Dystrophy, Muscular Dystrophy, Duchenne, Projections, Sales - occupational activity

Sarepta Therapeutics, ELEVIDYS, Duchenne Muscular Dystrophy (DMD), Gene Therapy, Clinical Trial Results, Sales Projections

Regenxbio Advances Duchenne Muscular Dystrophy Gene Therapy to Pivotal Studies, Potentially Challenging Sarepta’s Dominance

8 months ago talkbio0Tagged Approved, Biologics License Application, DMD, Duchenne, ELEVIDYS, gene therapy, Muscular Dystrophy, Muscular Dystrophy, Duchenne, Regenxbio, SLC6A8 Inhibitor RGX-202, United States Food and Drug Administration

Regenxbio, Sarepta Therapeutics, Duchenne Muscular Dystrophy (DMD), Gene Therapy, RGX-202, Elevidys, Pivotal Studies, Biologics License Application (BLA), FDA Approval

Sanofi Sues Sarepta Over US Patents for Gene Therapy Treatments

12 months ago talkbio0Tagged Duchenne, ELEVIDYS, gene therapy, Legal patent, Muscular Dystrophy, sanofi

Sanofi, Sarepta, Gene Therapy, Patents, Elevidys, Duchenne Muscular Dystrophy

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.