His parents Stephen and Jenny have asked Sheffield Children's NHS Foundation Trust to approve the use of drug givinostat, ...

"At every visit, I'm thinking about bone health, talking about bone health," says Alexandra Bonner, MD, of the Center for Pediatric Neurosciences at Cleveland Clinic. She emphasizes the imortance of a ...

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that starts in childhood and causes muscle weakness that worsens over time. There’s no cure for DMD, but new treatments have helped slow ...

The Food and Drug Administration Thursday approved the first gene therapy treatment for Duchenne Muscular Dystrophy, a progressive disorder that typically robs boys of their ability to walk around age ...

DALLAS, Oct. 17, 2025 /PRNewswire/ -- The Killian family, of Rockwall, Texas, are celebrating a fundraising milestone with their annual Sam's Night event to benefit Parent Project Muscular Dystrophy ...

A tiny, rust-colored therapy dog snuggled beneath the blanket as Ryan Richard lay on a reclining sofa in the living room of the apartment he shares with his wife, Jamie. The tubes of a breathing ...

The Swiss pharma is paying $12 billion for Avidity Biosciences and its three late-stage antibody oligonucleotide conjugates.