DMD
Avidity’s Del-zota Shows Promise in Duchenne Muscular Dystrophy Treatment
Del-zota, Duchenne muscular dystrophy (DMD), RNA therapeutics, exon 44 skipping, dystrophin production, creatine kinase reduction, safety profile
Entrada given go-ahead to test DMD therapy after two-year hold
Entrada, Muscular Dystrophy, Duchenne, DMD, United States Food and Drug Administration, Adult, Exons
Sarepta Therapeutics Reports Encouraging Two-Year Data for ELEVIDYS in Duchenne Muscular Dystrophy, Projecting $1 Billion in Sales
Sarepta Therapeutics, ELEVIDYS, Duchenne Muscular Dystrophy (DMD), Gene Therapy, Clinical Trial Results, Sales Projections
Regenxbio Advances Duchenne Muscular Dystrophy Gene Therapy to Pivotal Studies, Potentially Challenging Sarepta’s Dominance
Regenxbio, Sarepta Therapeutics, Duchenne Muscular Dystrophy (DMD), Gene Therapy, RGX-202, Elevidys, Pivotal Studies, Biologics License Application (BLA), FDA Approval
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
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.