Muscular dystrophies are a group of more than 30 genetic disorders characterized by progressive muscle weakness and wasting. These disorders are typically associated with defects in muscle ...
A retrospective cohort study found that, among 459 patients with DMD, constipation (32.5%) was the most common GI complication, followed by dysphagia (9.4%). Older age, lower body mass index, and use ...
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 ...
The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...
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Sarepta surges as it takes on Novartis' $12 billion muscular dystrophy buyout
Sarepta stock popped Wednesday after the company reported promising results for its gene-silencing tech in two forms of muscular dystrophy.
A new study led by researchers at Hospital for Special Surgery (HSS) suggests that currently available therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy (DMD), a ...
Duchenne therapy may be evolving from slowing disease to reversing it—with RNA approaches offering the prospect of restoring function. Ongoing research will determine if these advances can deliver ...
Genetic testing can confirm a muscular dystrophy (MD) diagnosis when symptoms and other tests already suggest MD. It also identifies specific gene mutations that can guide targeted treatment. Genetic ...
Sarepta Therapeutics’ report of a second fatality this year in a patient dosed with its Duchenne muscular dystrophy gene therapy raised the prospect it might have to pull the product from the market.
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 ...
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