A new treatment platform developed by researchers at the University of Texas MD Anderson Cancer Center was able to deliver ...

Health Canada Priority Review targets an expedited 180-day review period for givinostat, reflecting Italfarmaco’s commitment to Canadian DMD ...

Engineered extracellular vesicles delivering full-length DMD mRNA restored dystrophin and improved muscle strength and ...

GEn1E Lifesciences Inc., a Phase 2 clinical-stage biotechnology company advancing AI-driven precision medicines for ...

Panelists discuss how the pooled 3-year data for delandistrogene moxeparvovec show sustained motor function stabilization and less deterioration compared with natural history, providing confidence in ...

When Dean Burkin, Ph.D., first began studying Duchenne muscular dystrophy (DMD), the goal was straightforward and urgent: understand why muscles fail in this devastating disease and find a way to slow ...

New treatments and gene-based therapies are changing the outlook for those living with Duchenne Muscular Dystrophy, a rare ...

Experts discuss the landscape of treatment options for Duchenne muscular dystrophy, examine the benefits and risks of gene therapy and conventional steroid treatments, and emphasize the need for early ...

Duchenne muscular dystrophy (DMD) is a genetic disorder that mainly affects boys. DMD is usually diagnosed in early childhood and tends to worsen over time. DMD happens when a change (mutation) in the ...

Sarepta Therapeutics said it will update its prescribing information for Elevidys ® (delandistrogene moxeparvovec-rokl) after acknowledging the sudden death of a patient with Duchenne muscular ...

The patient in this case had suffered acute liver failure following the Elevidys infusion. Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy (DMD) who received Elevidys ...