Newborn screening is also cost-effective. Testing that takes just a few dollars per baby can save families and the health ...

After months of uncertainty and controversy, the FDA has given approval to Sarepta's Duchenne muscular dystrophy drug ... available for the rare genetic muscle-wasting disease in the ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

Duchenne muscular dystrophy (DMD) is the most common genetic muscle disorder, affecting 23,000 boys in the US each year. The disease results from a mutation in the gene that encodes dystrophin — the ...

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the latest ...

Suneel Ram’s needs gradually became greater after he was diagnosed at age 3 with Duchenne muscular dystrophy, a muscle-wasting genetic disorder that afflicts one in 5,000 boys. Today ...

However, there are some who appear lethargic due to muscle weakness in their bodies. This condition could be due to a congenital disorder called Duchenne muscular dystrophy ( DMD). DMD was first ...

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

This dystrophin transcript, theoretically, would exist in both muscle and the central nervous system, resulting in Duchenne muscular dystrophy in the former and neuronal dysfunction in the latter ...

Duchenne, the most common form of childhood muscular dystrophy, is a progressive and fatal disorder that results in the loss of muscle function, decline in respiratory and cardiac function and ...