Muscular Dystrophy, Duchenne – Page 3

FDA Threatens Action After Sarepta Refuses to Halt Elevidys Shipments

4 months ago talkbio0Tagged ELEVIDYS, gene therapy, Liver Failure, Market, Muscular Dystrophy, Duchenne, patient deaths, Regulatory Authority, United States Food and Drug Administration, Withdraw (activity)

FDA; Sarepta Therapeutics; Elevidys; gene therapy; market withdrawal; patient deaths; Duchenne muscular dystrophy; liver failure; regulatory authority

Sarepta Stands Ground Against HHS Request To Stop Elevidys Shipments

4 months ago talkbio0Tagged AAVrh74, CHARGE Syndrome, drug safety, ELEVIDYS, gene therapy, Muscular Dystrophy, Duchenne, patient deaths, Regulatory Action, Ships, Suspensions, United States Food and Drug Administration

Sarepta; Elevidys; FDA; HHS; gene therapy; Duchenne muscular dystrophy; drug safety; shipping suspension; patient deaths; AAVrh74; regulatory action

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

4 months 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 Rejects Capricor’s Cell Therapy for Duchenne Muscular Dystrophy

5 months ago talkbio0Tagged Capricor Therapeutics, Cardiomyopathies, Cell Therapy, Clinical Data, Complete, deramiocel, HOPE-3 trial, Muscular Dystrophy, Duchenne, Phase 3, Rejection, Response process, United States Food and Drug Administration

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

5 months ago talkbio0Tagged Clinical trial failure, Muscular Dystrophy, Duchenne, Otsuka Holdings, pizuglanstat, REACH-DMD study, Taiho, TAS-205

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

5 months 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

5 months 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

5 months 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

Regenxbio Reports Consistent One-Year Benefit for DMD Gene Therapy RGX-202

6 months ago talkbio0Tagged AFFINITY DUCHENNE trial, Biological Markers, Clinical Trials, Functional, gene therapy, Muscular Dystrophy, Duchenne, Phase III, Regenxbio, SLC6A8 Inhibitor 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

7 months ago talkbio0Tagged ENTR-601, exon, Muscular Dystrophy, Duchenne, Study on Hold, United States Food and Drug Administration

Duchenne muscular dystrophy (DMD), workforce reduction, ENTR-601-44, ENTR-601-45, FDA clinical hold, exon skipping therapy