Related coverage linked through entity extraction aliases.
May 12, 2026
Phase 1 data in 17 adults with Friedreich's ataxia cardiomyopathy showed AAVrh.10hFXN (LX2006) was generally well tolerated and showed signs of biological activity. The gene therapy is being advanced into a larger trial.
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.
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PTC Therapeutics has withdrawn its New Drug Application for Translarna (ataluren) for Duchenne muscular dystrophy after the FDA indicated the data were unlikely to meet approval thresholds,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.
Feb 13, 2026
PTC Therapeutics has withdrawn its New Drug Application resubmission for ataluren (Translarna) for nonsense mutation Duchenne muscular dystrophy after the FDA indicated the submission lacked substantial evidence of effectiveness.
