Norwegian documentary, The Remarkable Life of Ibelin, directed by Benjamin Ree, chronicles the life of Mats Steen, who had ...

Just as Pride Month ends, Disability Pride Month begins. The intersection between gender, sexuality, and disability is ever ...

A second Duchenne muscular dystrophy patient died after being treated with the gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said Sunday.

The one-time treatment is approved for children with a genetic variant of Duchenne’s muscular dystrophy, which causes weakness, loss of mobility and early death in males.

Duchenne muscular dystrophy affects approximately 15,000 people in the United States, primarily boys. The genetic disorder disrupts production of dystrophin, a protein essential for muscle function.

Second death linked to Sarepta Therapeutics treatment leaves Duchenne muscular dystrophy community reeling, Elevidys use halted in non-ambulatory patients.

When many people think of the Muscular Dystrophy Association (MDA), they probably think about the long-running telethon made famous by comedian Jerry Lewis. For decades, the telethon took over the ...

PepGen ends all work on Duchenne muscular dystrophy after lead asset fails to raise dystrophin levels ...

About Defeat Duchenne Canada Defeat Duchenne Canada is the country's only national charity dedicated to ending Duchenne muscular dystrophy.

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

The article highlights that neurologists and other clinicians treating people with Duchenne muscular dystrophy should be aware of the limitations of this treatment and the need to monitor and ...

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of ...