A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy ... DMD patients with DMD mutations amenable to exon 44 skipping ...

New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing severe muscle degeneration, also profoundly affects the brain, leading to ...

Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

The FDA cleared Sarepta to start dosing in a first-in-human clinical trial testing the gene therapy SRP-9005 in people with ...

Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%. A recent study ...

adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the ...