INDIANAPOLIS — A local mom is pushing for a rare and deadly disease to be added to Indiana's newborn screening panel. Duchenne muscular dystrophy affects at least 1 in 5,000 male births each year.

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is linked ...

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 ...

"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne Muscular Dystrophy Cara Lynn Shultz is a writer-reporter at PEOPLE. Her ...

Investigators based in Italy recently assessed the impacts of swallowing difficulties in a cohort of patients with DMD who presented to a single hospital with respiratory issues. The Swallowing ...

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 ...

A patient has died following treatment with Elevidys, the Sarepta Therapeutics product that is the only FDA-approved gene therapy for Duchenne muscular dystrophy, the company disclosed Tuesday. The ...

More than 30 rare muscular dystrophy types cause progressive muscle weakness from inherited gene mutations. Combined, they affect about 1 in 5,000 to 8,000 people. Common subtypes vary by age of onset ...

There is no cure for DMD, but treatments can slow the progression of the disease and help improve quality of life. Duchenne muscular dystrophy (DMD) is a chronic condition that causes a gradual loss ...