A mother's love drives her to pursue experimental treatment as two of her four children battle a rare genetic disease that ...

Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...

Please provide your email address to receive an email when new articles are posted on . The FDA has approved an abbreviated new drug application for a generic version ...

On March 27, 2024, Ann & Robert H. Lurie Children's Hospital of Chicago treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular ...

CureDuchenne Clinic within the Neuromuscular Program at CHOC Will Expand Access to Exceptional Care, Deliver Cutting-Edge Treatments, and Drive Innovative Research NEWPORT BEACH, Calif., March 3, 2025 ...

EPIDYS trial met primary endpoint demonstrating statistically and clinically meaningful treatment benefit in one of the largest DMD phase 3 trials to date Median follow-up of over 3 additional years ...

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 ...

Each week, this series shares MarionMade! stories of our many wonderful people, places, products and programs in the greater Marion community. To read more positive stories of Marion, or to share some ...

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 ...

—Older age, presence of scoliosis, and other factors were associated with lower predicted forced vital capacity in patients with Duchenne muscular dystrophy using non-invasive ventilation. Individuals ...

Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...