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

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 ...

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 ...

Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy, the biotech has closed a public offering that raised an impressive ...

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

While genetic testing could not confirm Duchenne muscular dystrophy, a muscle biopsy at UCLA did. "He could see that he was declining, and that it was getting worse," said Stephanie. Pediatric ...

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 ...

We identified a novel and significant association between Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder. The eight cases with both diagnoses presented ...

Chronic muscle inflammation in Duchenne muscular dystrophy (DMD) is driven by both infiltrating and resident macrophages, the latter stimulated by CSF-1 produced by fibro/adipogenic progenitors.