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 ...
The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...
Missing an important procedure because of a medical oversight causes columnist Betty Vertin to rethink caregiving for her sons with Duchenne.
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 ...
Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...
—Duchenne muscular dystrophy is a complicated chronic disease. This study adapted an algorithm and used disease stage criteria to present a detailed picture of its multiple aspects. Duchenne muscular ...
The FDA has officially limited the label of Sarepta Therapeutics’ Elevidys, putting an end to a whirlwind few months that saw the abrupt departure—and reinstatement—of top agency official Vinay Prasad ...
Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the deterioration of skeletal muscle in the disease. The use of ...
A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback