Duchenne
Major Clinical Trial Failures of 2024: Setbacks in Neuroscience and Beyond
Clinical trials, drug development, neuroscience, ALS, Alzheimer’s disease, schizophrenia, Duchenne muscular dystrophy, oncology
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
FDA Places Clinical Hold on PepGen’s Duchenne Muscular Dystrophy Trial
FDA, Clinical Hold, PepGen, Duchenne Muscular Dystrophy, PGN-EDO51, CONNECT2-EDO51
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
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
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
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
Sanofi Sues Sarepta Over US Patents for Gene Therapy Treatments
Sanofi, Sarepta, Gene Therapy, Patents, Elevidys, Duchenne Muscular Dystrophy
Sarepta Therapeutics: Next Steps After Full FDA Approval for Duchenne Muscular Dystrophy Gene Therapy
Sarepta Therapeutics, Duchenne muscular dystrophy, gene therapy, FDA approval, Elevidys, microdystrophin, ambulatory patients, non-ambulatory patients, pipeline development, rare diseases, genetic medicine.