It’s caused by mutations in the DMD gene, which carries the instructions for making the protein dystrophin. Approximately 70% to 80% of people with DMD inherit a genetic mutation that causes the ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...

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 ...

Duchenne muscular dystrophy (DMD) is the most common genetic muscle disorder, affecting 23,000 boys in the US each year. The disease results from a mutation in the gene that encodes dystrophin — the ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy (DMD) who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment. Elevidys is administered as ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin, a protein that is missing or defective in DMD patients. DMD is a form of ...

Opens in a new tab or window A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta ...

Two-year data showed significant functional improvements in patients treated with delandistrogene moxeparvovec compared to controls, despite initial trial endpoint failure. The treated group ...

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 ...

Sarepta’s portfolio consists of four approved therapies, all targeting DMD indications. Currently, Elevidys is the first and only one-shot gene therapy for DMD in the United States. Since its ...