News Related to dystrophin

Solid Biosciences Advances SGT-003 DMD Gene Therapy with FDA Alignment, Reports No Liver Toxicity

Mar 12, 2026

Solid Biosciences has secured FDA alignment on a registration pathway for its Duchenne muscular dystrophy gene therapy SGT-003, with 36 patients dosed to date showing no drug-induced liver injury, myocarditis, or other serious adverse events reported with competing therapies.

Precision BioSciences Receives FDA Fast Track Designation for PBGENE-DMD Gene Editing Therapy

Mar 09, 2026

Precision BioSciences announced FDA Fast Track designation for PBGENE-DMD, a first-in-class gene editing therapy for Duchenne muscular dystrophy targeting mutations in exons 45-55, representing up to 60% of DMD patients.

Chugai Launches ELEVIDYS as Japan's First Gene Therapy for Duchenne Muscular Dystrophy

Feb 28, 2026

Chugai Pharmaceutical launched ELEVIDYS in Japan as the first regenerative medical product for Duchenne muscular dystrophy following its listing on the National Health Insurance reimbursement price list on February 20, 2026.

PTC Therapeutics Withdraws Translarna NDA After FDA Feedback on Efficacy

Feb 12, 2026

PTC Therapeutics has withdrawn its New Drug Application resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy following FDA feedback that the data are unlikely to meet the threshold for substantial evidence of effectiveness.

PTC Therapeutics Withdraws FDA Application for Duchenne Drug Translarna After Decade-Long Effort

Feb 13, 2026

PTC Therapeutics has withdrawn its New Drug Application for Translarna (ataluren) for Duchenne muscular dystrophy after the FDA indicated the data were unlikely to meet approval thresholds, ending a pursuit spanning more than a decade.

PTC Therapeutics Withdraws Translarna NDA After FDA Cites Insufficient Efficacy Data

Feb 13, 2026

PTC Therapeutics withdrew its resubmitted new drug application for Translarna in Duchenne muscular dystrophy after the FDA indicated the data was unlikely to meet the threshold for substantial evidence of effectiveness, marking the third unsuccessful US approval attempt.