For several years, scientists have been pondering a question about a genetic disease called Charcot-Marie-Tooth (CMT) disease type 2D: how can different types of mutations, spread out across a gene, produce the same condition?
Jul 4, 2011
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Researchers at the National Institutes of Health and their colleagues have found that a toxic protein made by the body called DUX4 may be the cause of two very different rare genetic disorders. For patients who have facioscapulohumeral ...
Feb 17, 2023
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Researchers at the University of British Columbia's School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy.
Jun 29, 2022
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Each year, about 20,000 children are diagnosed with Duchenne muscular dystrophy, a rare genetic condition that causes progressive muscle weakness and other systemic damage.
Feb 18, 2022
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Researchers have discovered a new technique that overcomes one of the major challenges of stem cell therapy.
Jan 25, 2018
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Macrophages, a type of white blood cell involved in inflammation, readily take up a newly approved medication for Duchenne muscular dystrophy (DMD) and promote its sustained delivery to regenerating muscle fibers long after ...
Oct 16, 2017
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A new study from The Ottawa Hospital and the University of Ottawa is poised to completely change our understanding of Duchenne muscular dystrophy and pave the way for far more effective treatments.
Nov 16, 2015
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UT Southwestern Medical Center researchers successfully used a new gene editing method to correct the mutation that leads to Duchenne muscular dystrophy (DMD) in a mouse model of the condition.
Aug 14, 2014
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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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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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