When a Harford County mom noticed her son was moving slower than his siblings, she became concerned. After years of trying to ...

Sarepta Therapeutics (SRPT) added ~25% in the morning hours on Wednesday after the company posted initial data from Phase 1/2 ...

Sarepta stock popped Wednesday after the company reported promising results for its gene-silencing tech in two forms of ...

The big pharma company will no longer progress emugrobart to late-stage trials in FSHD and SMA due to a lack of efficacy.

The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

Interim observations from the Phase 2 TRAILHEAD study show continued improvement in handgrip strength, overall stability of ...

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 ...

Duchenne muscular dystrophy (DMD) is a genetic condition caused by a change, or mutation, in the DMD gene. This gene normally tells the body how to make dystrophin, a protein that helps protect ...

"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 ...

The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...

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 ...

When most people think about music venues, gas stations do not come to mind. But the organizers of the Muscular Dystrophy ...