Nine people around the world who have been living with unexplained, muscular dystrophy-like symptoms may now have an answer for what is causing the progressive muscle weakness in their body's mid-section, arms and legs.
Jul 17, 2023
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The Food and Drug Administration on Friday approved the first treatment for children and adults with spinal muscular atrophy, a rare genetic disorder marked by progressive muscle weakness that's the most common genetic cause ...
Dec 23, 2016
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A recent finding by medical geneticists sheds new light on how Facioscapulohumeral Muscular Dystrophy develops and how it might be treated. More commonly known as FSHD, the devastating disease affects both men and women.
Nov 11, 2012
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Investigators at Nationwide Children's Hospital, working with the DNA Sequencing Core Facility at the University of Utah, have developed an approach to newborn screening (NBS) for the life-threatening genetic disorder, Duchenne ...
Mar 19, 2012
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Researchers studying a rare genetic disorder that causes severe, progressive neurological problems in childhood have discovered insights into biological mechanisms that drive the disease, along with early clues that an amino ...
Feb 22, 2018
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People with myotonic dystrophy experience progressive muscle weakness and repeated episodes of painless muscle stiffness called myotonia.
Apr 13, 2023
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Researchers have launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease, hereditary inclusion body myopathy (HIBM). National Institutes of Health scientists ...
Sep 24, 2012
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New insights into certain muscle diseases, the filaminopathies, are reported by an international research team led by Dr. Rudolf Andre Kley of the RUB's University Hospital Bergmannsheil in the journal Brain. The scientists ...
Sep 25, 2012
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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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Sometimes known as Kennedy's disease, spinal and bulbar muscular atrophy (SBMA) is a rare inherited neuromuscular disorder characterized by slowly progressive muscle weakness and atrophy. Researchers have long considered ...
Apr 16, 2014
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