R1 calpain3-related is extremely rare, and its prenatal management is unclear. A primigravida in her late 30s with LGMD R1 calpain3-related was referred to our hospital during the first trimester.

A Sarepta Therapeutics gene therapy that failed its confirmatory test has now converted its accelerated FDA approval into a traditional one, expanding use of the treatment to a wider group of Duchenne ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

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 ...

Shares of  Regenxbio rose Monday in premarket trading after the company said its treatment for Duchenne muscular dystrophy delivered positive trial results, strengthening the case for a speedier ...

The X-linked neuromuscular disorder Duchenne muscular dystrophy (DMD), which historically has been diagnosed in boys between the ages of 3 and 6 years, has become an important candidate for newborn ...

Pathologic changes of the X-chromosome gene for dystrophin give rise to Duchenne muscular dystrophy (DMD), and dystrophin -- which minimizes muscle fiber loss due to sarcolemma contraction damage when ...

COLUMBUS, Ohio — Ohio is now the first state in the nation to begin screening newborns for Duchenne Muscular Dystrophy, Gov. Mike DeWine announced Monday. The provision was included in the state’s ...

A person may inherit the genetic changes responsible for muscular dystrophy. These genetic changes can also occur due to spontaneous genetic mutations. In either case, the disease is not preventable.

PHOENIX (AZFamily) —As we head toward the new year, the medical advances just keep coming for the muscular dystrophy community. New treatments and therapies are being approved to help families ...