In September, the Food and Drug Administration approved Exondys, a controversial treatment for Duchenne muscular dystrophy based on tenuous data from just 12 patients. The cover story in Chemical & Engineering News (C&EN), ...
Nov 16, 2016
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Progressively shortening telomeres—the protective caps on the end of chromosomes—may be responsible for the weakened, enlarged hearts that kill many sufferers of Duchenne muscular dystrophy, according to a study by researchers ...
Oct 31, 2016
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Duchenne is the most common and severe form of muscular dystrophy. Because of this genetic disease, one out of every 3,500 children spends their 12th birthday in a wheelchair. This disorder progressively leads to general ...
Oct 19, 2016
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A new paper, co-written by faculty at Binghamton University, State University of New York, increases the understanding of Duchenne muscular dystrophy (DMD)—one of the most common lethal genetic disorders—and points to ...
Oct 13, 2016
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A study published today by scientists at University of Massachusetts Medical School and the University of Alabama at Birmingham provides insight into the mechanism of action of the drug ataluren, which is showing promise ...
Oct 4, 2016
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Muscular dystrophy is a group of rare genetic diseases that cause progressive muscle weakness and deterioration. For decades, researchers have struggled to understand how the disease works and to find suitable treatments.
Oct 4, 2016
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The powerful US Food and Drug Administration (FDA) has given the green light to a drug developed by WA researchers Sue Fletcher and Steve Wilton for treating Duchenne muscular dystrophy.
Sep 21, 2016
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A research team led by the University of Oxford has found a promising treatment for degenerative disease spinal muscular atrophy (SMA), a leading genetic cause of child death.
Sep 20, 2016
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Federal regulators on Monday granted tentative approval to the first drug for muscular dystrophy, following an intense public campaign from patients and doctors who pushed for the largely unproven medication.
Sep 19, 2016
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Duchenne muscular dystrophy is a chronic disease causing severe muscle degeneration that is ultimately fatal. As the disease progresses, muscle precursor cells lose the ability to create new musclar tissue, leading to faster ...
Sep 12, 2016
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