Muscular Dystrophy, Duchenne
Analysts Uncover Nuances in Avidity’s DMD Data, Highlighting Promising Results
Avidity Biosciences, DMD, Duchenne Muscular Dystrophy, RNA therapy, phase 1/2 data, clinical trials, biotech, pharmaceuticals
NS Pharma’s Viltepso Fails Confirmatory Phase III Trial for Duchenne Muscular Dystrophy Treatment
Viltepso, NS Pharma, Duchenne muscular dystrophy, RACER53 study, viltolarsen, FDA approval, confirmatory trial, phase III trial, DMD treatment
EU Upholds Approval of PTC Therapeutics’ Duchenne Muscular Dystrophy Treatment
EU, PTC Therapeutics, Duchenne Muscular Dystrophy (DMD), treatment, approval, rejection, committee.
Translarna by PTC Therapeutics: Awaiting Another EU Regulatory Review for Muscular Dystrophy Treatment
PTC Therapeutics, Translarna, muscular dystrophy, EU review, regulatory approval, Duchenne muscular dystrophy (DMD), European Medicines Agency (EMA), ataluren, rare disease treatment.
Duchenne Muscular Dystrophy: Potential Inclusion in Newborn Screening Programs
Duchenne muscular dystrophy, newborn screening, medical experts, decision-making, early diagnosis, treatment options.
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.