Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort ...

The virus isn't harmful, but the gene it's carrying is known to put the brakes on the progression of DMD. The hope is that this therapy slows down Colton Belluzzo's disease, buying time for him to ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

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 ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin ... of this devastating disease. We look forward to sharing additional ...

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

Leerink Partners analysts pointed out in a note this morning that this 3.8-second improvement is greater than the 2.7-second benefit seen one year after taking Sarepta’s DMD gene therapy Elevidys.

The sector was also shaken by the death of a patient who was dosed with Sarepta Therapeutic’s DMD gene therapy Elevidys ... accelerated approval, Viltepso looks to be a promising therapy ...

Almost against the odds, Sarepta’s gene therapy for muscle-wasting disease Duchenne muscular dystrophy (DMD ... “We actively partner with the FDA to look for any way to speed the approval ...