Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells. Cold Spring Harbor Laboratory Professor Adrian Krainer leveraged ASO technology to develop the first FDA-approved treatment ...
Jul 14, 2022
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Researchers have successfully improved the ability of muscle to repair itself - by artificially increasing levels of the BMI1 gene in the muscle-specific stem cells of mice with muscular dystrophy.
Jan 15, 2015
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Researchers from Nationwide Children's Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with ...
Jun 15, 2020
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(Medical Xpress)—Facioscapulohumeral muscular dystrophy (FSHD) is a genetic disorder that causes the muscles of the upper body to waste away. It is unusual in that symptoms do not usually appear until sufferers are in their ...
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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A potential way to treat muscular dystrophy directly targets muscle repair instead of the underlying genetic defect that usually leads to the disease.
Sep 14, 2014
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Researchers have gained insight into how a toxic protein, known to trigger muscular dystrophy, could be deactivated—a pre-clinical discovery that could spearhead a treatment for the debilitating disease.
Sep 25, 2023
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(Medical Xpress) -- A team led by scientists at UCL, funded by the Medical Research Council (MRC) and AVI BioPharma, have made an important breakthrough in the development of a treatment for Duchenne Muscular Dystrophy (DMD).
Jul 25, 2011
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People who have the most common type of adult muscular dystrophy also have a higher risk of getting cancer, according to a paper published today in the Journal of the American Medical Association.
Dec 13, 2011
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Spinal muscular atrophy (SMA) is a genetic disease that affects motor neurons in the spinal cord, resulting in muscle atrophy and widespread weakness that eventually impair swallowing and breathing. A new study in the Journal ...
Jul 16, 2018
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