The study provides evidence of survival benefits among patients with Duchenne muscular dystrophy (DMD) receiving eteplirsen compared with the natural history of the condition. DMD is a genetic ...
Consensus directed medical therapy (CDMT) was used in 73.1% of DMD patients with moderate or severe left ventricular dysfunction, leaving nearly 30% without recommended treatment. Target doses were ...
Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Based on western blot testing, ...
Sarepta Therapeutics said it has temporarily suspended shipments of Elevidys ® (delandistrogene moxeparvovec-rokl) for infusion in non-ambulatory patients following the second death in three months of ...
Shares of Dyne Therapeutics DYN rose 10% on Wednesday after the company announced the initiation of the phase III FORZETTO study evaluating the safety and efficacy of zeleciment rostudirsen ...
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