A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a large-scale clinical ...
Apr 8, 2025
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New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing severe muscle degeneration, also profoundly affects the brain, leading to cognitive and behavioral ...
Mar 28, 2025
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Researchers at the Experimental and Clinical Research Center (ECRC), a joint institution of the Max Delbrück Center and Charité—Universitätsmedizin Berlin, have developed a promising gene-editing approach intended to ...
Jan 7, 2025
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A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1—the most prevalent form of adult-onset muscular dystrophy—provides insight into why patients develop fatty liver disease and display hypersensitivity ...
Oct 24, 2024
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Scientists at Sanford Burnham Prebys and Salk Institute for Biological Studies have uncovered a new role for a protein known for its role in the brain helping control feelings of hunger or satiety, as well as in the liver ...
Aug 2, 2024
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Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people ...
Jul 25, 2024
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A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those ...
Jul 17, 2024
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Biomedical engineers at Duke University have developed a new technique to better understand and test treatments for a group of extremely rare muscle disorders called dysferlinopathy or limb girdle muscular dystrophies 2B ...
Jun 21, 2024
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In an animal model of Duchenne muscular dystrophy, Brazilian researchers tested a therapy that combines photobiomodulation using laser light or light-emitting diodes (LEDs) with idebenone, an antioxidant compound investigated ...
Jun 14, 2024
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A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, or muscular dystrophies. This is shown in a new study carried out at Umeå University, Sweden, ...
Mar 6, 2024
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