The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...

The FDA has officially limited the label of Sarepta Therapeutics’ Elevidys, putting an end to a whirlwind few months that saw the abrupt departure—and reinstatement—of top agency official Vinay Prasad ...

People with Duchenne muscular dystrophy have a shorter-than-average life expectancy due to changes in muscles that affect breathing, heart activity, and other functions. However, treatment advances ...

Duchenne muscular dystrophy (DMD) results from changes in the DMD gene. Children may inherit DMD gene changes from their parents or have spontaneous gene changes. The DMD gene tells the body how to ...

MARYVILLE, Tenn. — Nine-year-old Will Leko loves Pokémon, swimming and video games. His favorite titles include Yoshi’s Crafted World and Super Mario Odyssey. But Will is also living with something ...

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 ...

Darren Ormiston, a 9-year-old boy with Duchenne muscular dystrophy, suddenly screamed and lept from his wheelchair, running to a doctor with his hands in the air. Darren wasn’t in distress, just ...

People with Duchenne muscular dystrophy (DMD) frequently live well into adulthood. Advances in treatment have significantly increased the life expectancy for this condition. The median life expectancy ...

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 ...

My name is Kelly Stacy. I am a mother of four sons, two of whom, Elijah and Kai, are still living with Duchenne muscular dystrophy, a rare and fatal disease that steals their ability to walk at the ...

Please provide your email address to receive an email when new articles are posted on . Cardiomyopathy in Duchenne muscular dystrophy possesses a unique pathophysiology. Integrated wall stress can be ...

The FDA lifted a voluntary hold on Elevidys, a Duchenne muscular dystrophy treatment, for ambulatory patients after two patient deaths in the non-ambulatory treatment group. Sarepta Therapeutics, the ...