Muscular Dystrophy, Duchenne – Page 2

Regenxbio Advances Duchenne Muscular Dystrophy Gene Therapy to Pivotal Studies, Potentially Challenging Sarepta’s Dominance

7 months ago talkbio0Tagged Approved, Biologics License Application, DMD, Duchenne, ELEVIDYS, gene therapy, Muscular Dystrophy, Muscular Dystrophy, Duchenne, Regenxbio, SLC6A8 Inhibitor RGX-202, United States Food and Drug Administration

Regenxbio, Sarepta Therapeutics, Duchenne Muscular Dystrophy (DMD), Gene Therapy, RGX-202, Elevidys, Pivotal Studies, Biologics License Application (BLA), FDA Approval

Sarepta Discontinues Development of SRP-5051 for Duchenne Muscular Dystrophy Due to Safety Concerns

7 months ago talkbio0Tagged Clinical Trials, concerns, Hypomagnesemia, Muscular Dystrophy, Duchenne, Safety, SRP-5051, United States Food and Drug Administration

Sarepta Therapeutics, SRP-5051, Duchenne muscular dystrophy, DMD, safety concerns, hypomagnesemia, FDA, clinical trials

Sarepta Therapeutics Halts Development of Vesleteplirsen, a Next-Generation Exon-Skipping Therapy for Duchenne Muscular Dystrophy

7 months ago talkbio0Tagged Duchenne, Exon-Skipping Therapy, Feedback - system communication, Landscapes, Muscular Dystrophy, Muscular Dystrophy, Duchenne, SRP-5051, United States Food and Drug Administration, Vesleteplirsen

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

8 months ago talkbio0Tagged cost-cutting measures, gene therapy, Muscular Dystrophy, Duchenne, North Carolina, Pfizer Layoffs, Trial failure

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

8 months ago talkbio0Tagged CHMP, Commission, Conditional, Decision, Drug Approval, European (ethnic group), Multiple Acyl Coenzyme A Dehydrogenase Deficiency, Muscular Dystrophy, Duchenne, regulatory, Translarna

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

9 months ago talkbio0Tagged Clinical Trials, DMD, Does skip, Duchenne, Dystrophin, exon 53, FORWARD-53, Gene Expression, Interim, Muscular Dystrophy, Muscular Dystrophy, Duchenne, Wave Life Sciences, WVE-N531

Wave Life Sciences, Duchenne Muscular Dystrophy, DMD, Exon 53 Skipping, WVE-N531, FORWARD-53 Clinical Trial, Interim Data, Muscle Health, Dystrophin Expression