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

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

After months of uncertainty and controversy, the FDA has given approval to Sarepta's Duchenne muscular dystrophy drug – even though it hasn't yet shown any proof it can slow progress of the ...

Duchenne muscular dystrophy (DMD) is a genetic disorder that ... DMD is an X-linked recessive disorder, a genetic pattern ...

She knew instantly that Charley had Duchenne muscular dystrophy (DMD ... for Duchennes,” says Dr. Louis Kunkel, Professor of Genetics and Pediatrics at Boston Children’s Hospital and Harvard ...

Oskoui, M., et al. (2025) Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus Report of the AAN Guidelines Subcommittee. Neurology . doi ...

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

Sarepta Therapeutics has decided not to continue the development of vesleteplirsen, a follow-up to its Duchenne muscular dystrophy (DMD) therapy Exondys 51. The drug – formerly known as SRP-5051 ...

The PJ Nicholoff Steroid Protocol has been instrumental in educating medical professionals on the unique care considerations for patients with Duchenne on long-term steroids who may have ...

"For nearly a decade, Sarepta has been a leader in advancing the treatment of Duchenne muscular dystrophy through innovative treatment options. With the approval of ELEVIDYS in Japan, children who ...