A new treatment for Huntington's disease – a deadly brain disorder – has successfully completed first-in-human trials. The drug lowered levels of the harmful huntingtin protein in the spinal fluid of patients who took ...
Dec 14, 2017
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In a recent paper published in the journal Skeletal Muscle, a Saint Louis University researcher reports success in identifying new drug targets that potentially could slow or halt the progression of a form muscular dystrophy, ...
Nov 7, 2017
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Motor neurons are the nerves that send impulses to the muscles to generate movement. Damage of these neurons can cause very diverse diseases, for example spinal muscular atrophy in children or adult amyotrophic lateral sclerosis.
Nov 2, 2017
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Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes ...
Oct 3, 2017
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The ability to cheaply and quickly sequence entire genomes is changing the way diseases are identified and treated. But it is also likely to change the way we make some of the most important and personal decisions of our ...
Aug 2, 2017
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Duchenne muscular dystrophy is a rapidly progressive disease that causes whole-body muscle weakness and atrophy due to deficiency in a protein called dystrophin. Researchers at the University of Missouri, National Center ...
Jul 27, 2017
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Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy—a type of muscular dystrophy that affects the skeletal muscles—have found that ...
Jun 8, 2017
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Heart muscle is one of the least renewable tissues in the body, which is one of the reasons that heart disease is the leading cause of death for both men and women in the United States, according to the Centers for Disease ...
Jun 5, 2017
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Glucocorticoid steroids are used to treat a variety of conditions, including chronic inflammatory diseases and autoimmune diseases. Their effects on muscle recovery and repair make them particularly effective therapies for ...
May 8, 2017
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Two papers published today by the Journal of the Royal Society of Medicine, debate gene editing and the health of future generations. Stage and screen actress Kiruna Stamell, who has a rare form of dwarfism, proposes that ...
Apr 27, 2017
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