Muscular Dystrophy, Duchenne

FDA OKs Sarepta Study of New Immune Suppression Approach with Elevidys; Gilead Acquires Preclinical TREX1 Program

2 days ago talkbio0Tagged acute liver injury, ELEVIDYS, gene therapy, Gilead, Muscular Dystrophy, Duchenne, sirolimus, Therapeutic immunosuppression, TREX1 gene, United States Food and Drug Administration

FDA; Sarepta; Elevidys; immunosuppression; Duchenne muscular dystrophy; Gilead; TREX1; gene therapy; sirolimus; acute liver injury

FDA Imposes Boxed Warning and Restricts Use of Sarepta’s Elevidys Gene Therapy for Duchenne Muscular Dystrophy

2 weeks ago talkbio0Tagged Black Box Warning, ELEVIDYS, gene therapy, Indication restriction, Liver Failure, Acute, Muscular Dystrophy, Duchenne, Post-marketing study, Safety labeling, United States Food and Drug Administration

Elevidys; Sarepta Therapeutics; Duchenne muscular dystrophy; FDA; Boxed warning; Gene therapy; Acute liver failure; Safety labeling; Indication restriction; Post-marketing study

Sarepta’s Duchenne Muscular Dystrophy Drugs Fail Confirmatory Trial, Company Pursues Full FDA Approval Regardless

3 weeks ago talkbio0Tagged Accelerated, AMONDYS, Approved, Clinical trial failure, Confirmatory Trial, ELEVIDYS, Muscular Dystrophy, Duchenne, Statistical Significance, United States Food and Drug Administration

Sarepta Therapeutics; Duchenne muscular dystrophy; confirmatory trial; AMONDYS 45; VYONDYS 53; Elevidys; FDA approval; clinical trial failure; accelerated approval; statistical significance

Dyne Therapeutics’ z-rostudirsen Shows Durable Efficacy, Supporting Positive Regulatory Outlook

4 weeks ago talkbio0Tagged Accelerated, Approved, Biological Markers, Clinical Trials, DELIVER (veterinary product), Dyne Therapeutics, Effectiveness, Functional improvement, Muscular Dystrophy, Duchenne, regulatory approval, z-rostudirsen

Dyne Therapeutics; z-rostudirsen; DELIVER trial; Duchenne muscular dystrophy (DMD); durable efficacy; regulatory approval; biomarkers; functional improvement; accelerated approval

Novartis to Acquire RNA Drugmaker Avidity Biosciences in $12 Billion Deal

1 month ago talkbio0Tagged 2026, Acquisition, AOC, Avidity Biosciences, Closed, Muscular Dystrophy, Duchenne, Muscular Dystrophy, Facioscapulohumeral, MYOTONIC DYSTROPHY 1, Neuromuscular Diseases, Novartis, Platform, RNA therapeutics, SpinCo ', xRNA

Novartis; Avidity Biosciences; acquisition; RNA therapeutics; neuromuscular diseases; Duchenne muscular dystrophy; myotonic dystrophy type 1; facioscapulohumeral muscular dystrophy; AOC platform; SpinCo; biotech; xRNA strategy; 2026 closing

Satellos Advances Duchenne Pill Trial in Children; Idorsia Raises $81 Million

2 months ago talkbio0Tagged Child, Fund Raising, Idorsia, Indonesian language, Muscular Dystrophy, Duchenne, oral small-molecule therapy, Phase 2 clinical trial, SAT-3247, Satellos, Submission, United States Food and Drug Administration

Satellos; Duchenne muscular dystrophy; SAT-3247; Phase 2 clinical trial; children; FDA IND submission; oral small-molecule therapy; Idorsia; fundraising; biotech

Avidity Touts Functional Improvements for DMD Therapy, Clearing Way to FDA

3 months ago talkbio0Tagged Approved, Avidity, Biosciences, Blackfoot language, Breakthrough Therapy, Clinical Trials, Functional, Improvements, Muscular Dystrophy, Duchenne, skipping, Submission, United States Food and Drug Administration

Avidity Biosciences; del-zota; DMD; Duchenne muscular dystrophy; FDA approval; functional improvements; exon 44 skipping; Breakthrough Therapy; clinical trial; BLA submission

Capricor Claims FDA Misinterpreted Efficacy Data Leading to Rejection of DMD Therapy

3 months ago talkbio0Tagged Capricor Therapeutics, Cell Therapy, Clinical Trials, data misinterpretation, deramiocel, Effectiveness, Endpoints, HOPE-2 trial, Muscular Dystrophy, Duchenne, Non-Parametric Test, Rejection, Statistical Significance, United States Food and Drug Administration

Capricor Therapeutics; FDA rejection; deramiocel; Duchenne muscular dystrophy (DMD); efficacy data misinterpretation; clinical trial endpoints; HOPE-2 trial; non-parametric test; statistical significance; cell therapy

Sarepta Pursues Efficiency Measures Amid $1 Billion Debt Due in 2027

4 months ago talkbio0Tagged 2025, debt, decline, Efficiency, ELEVIDYS, Financial cost, financial results, Financial savings, Measures, Muscular Dystrophy, Duchenne, restructuring, United States Food and Drug Administration

Sarepta Therapeutics; debt 2027; efficiency measures; ELEVIDYS; cost savings; restructuring; financial results 2025; FDA; Duchenne muscular dystrophy; stock decline

Untangling Sarepta’s Gene Therapy Fallout and Trust Deficit

4 months ago talkbio0Tagged AAVrh74 vector, clinical holds, ELEVIDYS, gene therapy, Muscular Dystrophies, Limb-Girdle, Muscular Dystrophy, Duchenne, patient deaths, Regulatory Action, United States Food and Drug Administration

Sarepta Therapeutics; gene therapy; Elevidys; Duchenne muscular dystrophy; limb-girdle muscular dystrophy; patient deaths; FDA; AAVrh74 vector; regulatory action; clinical holds; transparency