As it gears up to submit an approval application next year, Wave Life Sciences has presented fresh phase 2 data showing its ...

Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a ...

Amid readouts from the Muscular Dystrophy Association’s annual meeting, the Clinical Trials Arena evaluates five Duchenne ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death ...

This dystrophin gene correction approach which ... Since up to 60% of DMD cases are caused by defects between exons 45 and 55, this approach is more broadly applicable for the majority of DMD ...

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 ...

One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech said (PDF) Tuesday. The patient suffered ...

Avidity Biosciences’ Phase I/II trial of ribonucleic acid (RNA) therapy, del-zota, has increased dystrophin in patients with Duchenne muscular dystrophy (DMD). Results from the company’s ...