Duchenne
FDA Expands Approval of Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy to Include Older Boys and Non-Ambulatory Patients
FDA, Sarepta, Duchenne Muscular Dystrophy, Gene Therapy, Elevidys, Ambulatory, Non-Ambulatory, Expanded Approval
NS Pharma’s Duchenne Muscular Dystrophy Therapy Viltepso Fails Confirmatory Trial
NS Pharma, Viltepso, Duchenne Muscular Dystrophy, Confirmatory Trial, Phase III, RACER53 Study, FDA Approval
EU Upholds Approval of PTC Therapeutics’ Duchenne Muscular Dystrophy Treatment
EU, PTC Therapeutics, Duchenne Muscular Dystrophy (DMD), treatment, approval, rejection, committee.
Pfizer Temporarily Suspends Dosing in Advanced Duchenne Muscular Dystrophy (DMD) Trial After Patient Fatality
Pfizer, Duchenne Muscular Dystrophy (DMD), clinical trial, dosing halt, patient death, late-stage study, temporary suspension, safety concerns, investigation.
Controversy Surrounds FDA’s Fast-Track Approval of Sarepta’s Duchenne Muscular Dystrophy Gene Therapy
FDA, Sarepta Therapeutics, Duchenne Muscular Dystrophy (DMD), gene therapy, accelerated approval, controversy, clinical trials, efficacy, safety, patient advocacy, regulatory process.