The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with ...

The colorful threads in this image are muscle fibers. This spotted rainbow illustrates a potential gene therapy approach to treating Duchenne muscular dystrophy (DMD) – a rare, debilitating, and fatal ...

An abnormality in the DMD gene causes Duchenne muscular dystrophy. The DMD gene produces dystrophin, a protein that maintains the structure of muscle cells. People with DMD have mutations (changes ...

Duchenne muscular dystrophy (DMD) is a fatal genetic disorder caused by mutations in the DMD gene. Here, the authors develop MyoAAV-UA, a compact utrophin activator, as a promising universal ...

Without functional dystrophin to support muscle strength and stability, muscle fibers are easily damaged. Duchenne muscular dystrophy results from mutations in the DMD gene that encodes the dystrophin ...

DMD is a rare genetic disorder caused by ... the development of therapies for monogenic diseases - Successful human trials of gene transfer in the liver for hemophilia A and B, in the eye for ...

DMD is a rare genetic disorder caused by ... the development of therapies for monogenic diseases - Successful human trials of gene transfer in the liver for hemophilia A and B, in the eye for ...

Once the centre’s investigators were ready to launch first-in-human studies ... diseases like SMA and DMD start to affect babies in utero, we wanted to evaluate gene therapies for these patients ...

SGT-003 gene therapy showed significant microdystrophin expression and muscle integrity improvements in DMD patients, with no serious adverse events reported. The novel AAV vector demonstrated ...

Duchenne muscular dystrophy is a genetic disorder caused by mutations in the DMD gene, resulting in a lack of the protein dystrophin. This deficiency leads to progressive muscle weakness and loss ...

Solid Biosciences has surged back into the Duchenne muscular dystrophy (DMD) gene therapy race. The biotech reported expectation-busting phase 1 data on its next-generation candidate, sending its ...