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 ...

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 ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a ...

A Sarepta Therapeutics gene therapy that failed its confirmatory test has now converted its accelerated FDA approval into a traditional one, expanding use of the treatment to a wider group of Duchenne ...

Pathologic changes of the X-chromosome gene for dystrophin give rise to Duchenne muscular dystrophy (DMD), and dystrophin -- which minimizes muscle fiber loss due to sarcolemma contraction damage when ...

Werner is CEO of Alltrna and CEO-partner at Flagship Pioneering. Duchenne muscular dystrophy has seen more progress in the past 15 months (give or take) than in the last couple of decades combined. In ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Ohio will become the first state in the U.S. to screen newborns for a severe form of muscle disease called Duchenne muscular dystrophy (DMD), the governor’s office recently announced. DMD is the most ...

COLUMBUS, Ohio — Ohio will become the first state in the nation to begin screening all newborn babies for Duchenne Muscular Dystrophy, Gov. Mike DeWine announced Monday. DMD is characterized by ...