Muscular Dystrophy, Duchenne
Sarepta Therapeutics Halts Development of Vesleteplirsen, a Next-Generation Exon-Skipping Therapy for Duchenne Muscular Dystrophy
Sarepta Therapeutics, Vesleteplirsen (SRP-5051), Duchenne Muscular Dystrophy (DMD), Exon-Skipping Therapy, FDA Feedback, Therapeutic Landscape
Pfizer Announces Additional Layoffs in North Carolina Following DMD Trial Setback
Pfizer layoffs, North Carolina, Duchenne muscular dystrophy (DMD), gene therapy trial failure, cost-cutting measures
European Commission’s Reconsideration of Translarna’s Marketing Authorization: A Critical Turning Point for Duchenne Muscular Dystrophy Treatment
Translarna, Duchenne muscular dystrophy, European Commission, EMA, CHMP, conditional marketing authorization, drug approval, regulatory decisions
Wave Life Sciences Reports Positive Interim Data for Duchenne Muscular Dystrophy Treatment
Wave Life Sciences, Duchenne Muscular Dystrophy, DMD, Exon 53 Skipping, WVE-N531, FORWARD-53 Clinical Trial, Interim Data, Muscle Health, Dystrophin Expression
Capricor Secures $35M Deal for European Rights to DMD Therapy Ahead of FDA Application
Capricor Therapeutics, Duchenne muscular dystrophy (DMD), cell therapy, FDA application, European rights, $35M deal
Dyne Therapeutics Faces Setbacks Despite Promising DMD Study Results
Dyne Therapeutics, Duchenne Muscular Dystrophy (DMD), DELIVER trial, DYNE-251, adverse events, executive departures
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.