Scientists at Cold Spring Harbor Laboratory (CSHL) have identified a therapeutic RNA molecule that corrects the error in genetic processing that leads to familial dysautonomia, a rare inherited neurodegenerative disorder. ...
Apr 30, 2018
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A study of nearly half a million people has revealed that muscular strength, measured by handgrip, is an indication of how healthy our brains are.
Apr 19, 2018
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A preliminary study suggests that an investigational drug may help increase protein levels in babies with spinal muscular atrophy. The open-label study is released today and will be presented at the American Academy of Neurology's ...
Apr 18, 2018
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A major step forward has been made by researchers at the University of St Andrews towards finding new treatments for Spinal Muscular Atrophy (SMA), the commonest genetic cause of infant death.
Apr 18, 2018
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Swiss pharmaceuticals giant Novartis announced Monday it will buy US gene therapy company AveXis for $8.7 billion (7.1 billion euros).
Apr 9, 2018
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After a progressive weakening of the muscles takes away their motor skills, and then their abilities to stand and walk, most males with Duchenne muscular dystrophy die of heart and respiratory failure in their 20s.
Apr 6, 2018
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Gene therapy has shown promise in treating inherited genetic diseases, but a major issue that has frustrated scientists remains: Replacing a "bad" gene with a healthy one often is a short-lived fix. Typically, the healthy ...
Apr 2, 2018
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Zebrafish, roundworms, fruit flies, mice, rats, rabbits, dogs, cats, pigs, and monkeys provide steppingstones to clinical trials to evaluate new treatments for people. The value of animal studies continues, even after a new ...
Mar 30, 2018
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Using fruit flies, Texas A&M AgriLife Research scientists may be one step closer to better understanding the pathological mechanisms of muscular dystrophy.
Mar 28, 2018
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A drug that showed promise in clinical trials for Duchenne Muscular Dystrophy (DMD) decades ago is back under the microscope and has now been shown to reduce muscle wasting in mice.
Mar 20, 2018
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