gene therapy – Page 2

Vinay Prasad Departs FDA Amid Conservative Criticism and Sarepta Gene Therapy Controversy

3 weeks ago talkbio0Tagged CBER, conservative criticism, departure - ActRelationshipType, Drug Approval, gene therapy, Muscular Dystrophy, Duchenne, Prasad, Transplant Registry Unified Management Program, United States Food and Drug Administration, Vinay

Vinay Prasad; FDA; departure; Sarepta Therapeutics; gene therapy; Duchenne muscular dystrophy; conservative criticism; CBER; drug approval controversy; Trump administration

FDA Reverses Course, Allows Sarepta to Resume Duchenne Gene Therapy Shipments

4 weeks ago talkbio0Tagged ELEVIDYS, gene therapy, Muscular Dystrophy, Duchenne, Pauses, Resumption, Safety, shipment, United States Food and Drug Administration

FDA; Sarepta Therapeutics; Duchenne muscular dystrophy; Elevidys; gene therapy; safety pause; shipment resumption

FDA Investigates Elevidys Death; Sarepta and Roche Deny Link to Gene Therapy

4 weeks ago talkbio0Tagged Brazil, ELEVIDYS, gene therapy, Market, Muscular Dystrophy, Duchenne, Patient Death, Roche (company), Safety, Suspensions, United States Food and Drug Administration

FDA; Elevidys; Sarepta Therapeutics; Roche; gene therapy; Duchenne muscular dystrophy; patient death; Brazil; market suspension; safety concerns

CHMP Recommends Against Approval of Elevidys Gene Therapy in Europe, Dealing Blow to Sarepta and Roche

4 weeks ago talkbio0Tagged CHMP, ELEVIDYS, EMBARK trial, Europe, gene therapy, Multiple Acyl Coenzyme A Dehydrogenase Deficiency, Muscular Dystrophy, Duchenne, negative opinion, Roche (company)

CHMP; Elevidys; Sarepta; Roche; gene therapy; Duchenne muscular dystrophy; EMA; negative opinion; Europe; EMBARK trial

FDA Official Says Sarepta Faces Major Hurdles to Reintroduce DMD Therapy Elevidys

4 weeks ago talkbio0Tagged approval status, CBER, concerns, ELEVIDYS, gene therapy, Muscular Dystrophy, Duchenne, Pauses, Safety, Scientific Study, shipment, United States Food and Drug Administration

Sarepta; Elevidys; FDA; Duchenne muscular dystrophy; gene therapy; CBER; safety concerns; shipment pause; new studies; approval status

Roche Halts Elevidys Distribution in Several Countries Following Sarepta and FDA Safety Concerns

1 month ago talkbio0Tagged concerns, ELEVIDYS, gene therapy, halt, Muscular Dystrophy, Duchenne, patient deaths, Roche (company), Safety, shipment, United States Food and Drug Administration

Roche; Sarepta Therapeutics; Elevidys; Duchenne muscular dystrophy; gene therapy; FDA; shipment halt; safety concerns; patient deaths

Sarepta Halts US Shipments of Duchenne Therapy Elevidys After FDA Pressure

1 month ago talkbio0Tagged concerns, DMD, ELEVIDYS, gene therapy, Muscular Dystrophy, Duchenne, patient deaths, Safety, shipment, Suspensions, United States Food and Drug Administration

Sarepta Therapeutics; Elevidys; Duchenne muscular dystrophy (DMD); FDA; gene therapy; safety concerns; shipment suspension; patient deaths

Sarepta Therapeutics Faces FDA Crackdown after Third Patient Death; George Tidmarsh Named Top FDA Drug Regulator; Multiple Drugs Rejected; Pharma Invests Billions in US Manufacturing

1 month ago talkbio0Tagged drug rejection, ELEVIDYS, gene therapy, George Tidmarsh, Liver Failure, Acute, pharmaceutical manufacturing, reshoring, Study on Hold, United States Food and Drug Administration

Sarepta Therapeutics; FDA; gene therapy; Elevidys; acute liver failure; clinical hold; George Tidmarsh; drug rejection; reshoring; pharmaceutical manufacturing

Lack of Transparency Tarnishes Sarepta’s Sheen as Patient Deaths Trigger FDA Battle

1 month ago talkbio0Tagged ELEVIDYS, gene therapy, lack of transparency, Market, Muscular Dystrophies, Limb-Girdle, Muscular Dystrophy, Duchenne, patient deaths, Regulatory Action, United States Food and Drug Administration, Withdraw (activity)

Sarepta Therapeutics; Elevidys; patient deaths; FDA; lack of transparency; gene therapy; market withdrawal; regulatory action; limb-girdle muscular dystrophy (LGMD); Duchenne muscular dystrophy

Sarepta, bowing to FDA pressure, pauses shipments of Duchenne gene therapy Elevidys

1 month ago talkbio0Tagged ELEVIDYS, gene therapy, Muscular Dystrophy, Duchenne, patient deaths, Pauses, Safety, shipment, United States Food and Drug Administration

Sarepta Therapeutics; FDA; Elevidys; Duchenne muscular dystrophy; gene therapy; shipment pause; patient deaths; safety concerns