More than 30 types of muscular dystrophy have been identified. They all cause problems with movement due to muscle protein defects caused by genetic mutations. Muscular dystrophy is a group of ...
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 ...
Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...
Genetic testing, often via a blood sample, can confirm a muscular dystrophy diagnosis. It can also identify specific gene mutations to help guide treatment and determine whether or not someone is a ...
Facioscapulohumeral muscular dystrophy (FSHD) is a genetic condition that can lead to the slow deterioration of muscles, predominantly in the face, shoulder blades, and upper arms. Facioscapulohumeral ...
A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in ...
Researchers from Suzhou Genassist Therapeutics Co. Ltd. have developed a novel canine model of Duchenne muscular dystrophy (DMD), expected to be more predictive of disease pathogenesis and treatment ...
The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...
—Starting cardiac medications, using the proper timing and dosing, to optimize the efficacy of therapies and improve patient outcomes, is key to cardiac care for patients with Duchenne muscular ...
Muscular dystrophy (MD) is a group of genetic diseases that cause your muscles to progressively weaken and degenerate. There are several types of MD, each with its own symptoms, but they all involve ...