When you're diagnosing a child with Duchenne muscular dystrophy, says Alexandra Bonner, MD, of Cleveland Clinic, it's important to not only determine if she's a carrier of the gene mutation that ...

The Utah Program for Inherited Neuromuscular Disorders (UPIN) improves the lives of men, women, and children with inherited nerve or muscle disorders through cutting-edge, multidisciplinary care and ...

Vamorolone is approved and marketed as AGAMREE® for the treatment of Duchenne Muscular Dystrophy (DMD) in the US, European Union, UK and ChinaNew ...

Gene therapy may have the potential to cure Duchenne muscular dystrophy, but early results from clinical trials have not been as promising as researchers hoped. Research is ongoing. Duchenne muscular ...

Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...

Scientists have created a new gene therapy for Duchenne muscular dystrophy (DMD) that may not only help stop the disease in DMD patients, but might also help restore their damaged muscles in the ...

Oculopharyngeal muscular dystrophy (OPMD) is a rare inherited disorder that causes weakness in the eye and throat muscles. It may lead to drooping eyelids and swallowing difficulties. Muscular ...

A person may inherit the genetic changes responsible for muscular dystrophy. These genetic changes can also occur due to spontaneous genetic mutations. In either case, the disease is not preventable.

Duchenne therapy may be evolving from slowing disease to reversing it—with RNA approaches offering the prospect of restoring function. Ongoing research will determine if these advances can deliver ...

Fakeeh University Hospital is emerging as a global destination for advanced gene therapy, delivering highly specialised ...