"The most apparent take-away from the study findings is that a model of developmental therapy focusing on frequency, intensity, and variability of activity, exercise, and self-driven exploration may ...
COLUMBUS, Ohio (WCMH) – It’s being called a pioneering event that holds great promise for children who have Duchenne muscular dystrophy. After decades of research, animal studies, and human trials, ...
The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment's ability to help ...
COLORADO SPRINGS, Colo. (KRDO) -- A groundbreaking gene therapy was recently approved by the U.S. Food and Drug Administration to treat the most common form of muscular dystrophy in children, Duchenne ...
In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene ...
Waking up, hopping out of the bed, and stumbling to the kitchen for a cup of coffee: It’s an everyday routine most people don’t think twice about. But for children with spinal muscular atrophy, simply ...