Groundbreaking gene therapy offers hope for a boy with fatal muscular dystrophy, potentially slowing disease progression and ...

Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients ...

Muscle weakness has the biggest impact on patients’ ability to walk, but other factors matter too, researchers found.

Data from the EMBARK trial suggest patients may have functional improvements taking the gene therapy between 8 and 9 years old.

Since Elevidys' accelerated approval in 2023, experts have been clamoring for more data, particularly in older and ...

Duchenne Muscular Dystrophy, or DMD, is a rare and fatal genetic disease that primarily affects boys, slowly robbing them of ...

The approval allows the DMD gene therapy to be sold in Japan for up to seven years, but more trials will be needed for full ...

Capricor Therapeutics saw its shares fall 13% by the end of trading Monday as its investigational cell therapy deramiocel for ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.

Sarepta has submitted a request to the FDA to include the death case in Elevidys’ label, and the agency plans to review the ...

Elevidys is a prescription gene therapy developed by Sarepta Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). It targets the root cause of the disease by delivering a shortened but ...