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

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 ...

Duchenne muscular dystrophy (DMD) is perhaps the best known of the congenital muscular dystrophies. It is an X-linked disorder that is invariably progressive and fatal, affecting 1 in 3500 newborn ...

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 ...

Cas12Max-based gene editing therapy for Duchenne muscular dystrophy, one of the most severe forms of the inherited muscular dystrophies that affects primarily boys. Huidagene Therapeutics and its ...

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 ...

The 16-year-old’s infectious smile and easy charm have won over everyone from community members to politicians in both Indianapolis and Washington, D.C. But behind his charisma is a powerful cause: a ...