Muscular Dystrophy, Duchenne

Regenxbio Reports Consistent One-Year Benefit for DMD Gene Therapy RGX-202

2 days ago talkbio0Tagged AFFINITY DUCHENNE trial, Biological Markers, Clinical Trials, Functional, gene therapy, Muscular Dystrophy, Duchenne, Phase III, Regenxbio, SLC6A8 Inhibitor RGX-202

Regenxbio; DMD; Duchenne muscular dystrophy; RGX-202; gene therapy; AFFINITY DUCHENNE trial; clinical trial; microdystrophin; biomarker; phase I/II; functional data

Entrada tightens focus on Duchenne, axes 20% of staff

1 month ago talkbio0Tagged ENTR-601, exon, Muscular Dystrophy, Duchenne, Study on Hold, United States Food and Drug Administration

Duchenne muscular dystrophy (DMD), workforce reduction, ENTR-601-44, ENTR-601-45, FDA clinical hold, exon skipping therapy

Biotech Restructuring and European Regulatory Setbacks

2 months ago talkbio0Tagged Commission, European (ethnic group), factor IX, LifeMine, Muscular Dystrophy, Duchenne, regulatory, Translarna

LifeMine Therapeutics, layoffs, PTC Therapeutics, Translarna, Duchenne muscular dystrophy, European Commission, regulatory decision

Wave Life Sciences’ WVE-N531 Shows Promise in Duchenne Muscular Dystrophy Treatment

2 months ago talkbio0Tagged Approved, Dystrophin, Gene Expression, Health Improvement, Muscle Tissue, Muscular Dystrophy, Duchenne, Phase 2 Clinical Trials, skipping, United States Food and Drug Administration, WVE-N531

Duchenne muscular dystrophy (DMD), WVE-N531, exon 53 skipping, Phase II trial, dystrophin expression, muscle health improvement, FDA approval

Sarepta Reports Death of DMD Patient Following Elevidys Gene Therapy Treatment

3 months ago talkbio0Tagged acute, ELEVIDYS, gene therapy, Liver Failure, Muscular Dystrophy, Duchenne

Sarepta, Elevidys, gene therapy, Duchenne muscular dystrophy, patient death, acute liver failure

Avidity’s Del-zota Shows Promise in Duchenne Muscular Dystrophy Treatment

3 months ago talkbio0Tagged Creatine Kinase, Del-zota, DMD, Dystrophin, exon, Muscular Dystrophy, Duchenne, production, RNA, safety profile, skipping

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

3 months ago talkbio0Tagged Adult, DMD, Entrada, Exons, Muscular Dystrophy, Duchenne, United States Food and Drug Administration

Entrada, Muscular Dystrophy, Duchenne, DMD, United States Food and Drug Administration, Adult, Exons

FDA Lifts Clinical Hold on Entrada’s Duchenne Muscular Dystrophy Drug ENTR-601-44 After Two Years

3 months ago talkbio0Tagged ELEVATE-44, ENTR-601, Entrada, exon, Muscular Dystrophy, Duchenne, Phase 1b Trial, skipping, Study on Hold, United States Food and Drug Administration

Duchenne muscular dystrophy, ENTR-601-44, Entrada Therapeutics, FDA, clinical hold, exon 44 skipping, ELEVATE-44-102, Phase 1b trial

Solid Biosciences’ DMD Gene Therapy Shows Promising Early Results, Driving Stock Surge

4 months ago talkbio0Tagged Clinical Trials, gene therapy, Muscular Dystrophy, Duchenne, SGT-003, Solid Biosciences, surge

Duchenne muscular dystrophy (DMD), gene therapy, SGT-003, microdystrophin, clinical trial, stock surge, Solid Biosciences

Sarepta Therapeutics Reports Encouraging Two-Year Data for ELEVIDYS in Duchenne Muscular Dystrophy, Projecting $1 Billion in Sales

4 months ago talkbio0Tagged Clinical Trials, DMD, Duchenne, ELEVIDYS, gene therapy, Muscular Dystrophy, Muscular Dystrophy, Duchenne, Projections, Sales - occupational activity

Sarepta Therapeutics, ELEVIDYS, Duchenne Muscular Dystrophy (DMD), Gene Therapy, Clinical Trial Results, Sales Projections