MeSH tree: C16.320.577.300
A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular dystrophy, according to new research from the Smidt Heart Institute at Cedars-Sinai.
Aug 4, 2025
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The U.S. Food and Drug Administration's (FDA) top vaccine official is stepping down after just three months in a role that upset drug companies, patient groups and some political leaders.
Jul 31, 2025
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New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated that inhibiting GLUD1 ...
Jul 16, 2025
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Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular ...
Jul 15, 2025
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To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued ...
May 14, 2025
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The U.S. Food and Drug Administration (FDA) has chosen Dr. Vinay Prasad, a professor at the University of California-San Francisco, to lead its Center for Biologics Evaluation and Research.
May 7, 2025
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A new study led by researchers at the Hospital for Special Surgery (HSS) suggests that currently available therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy (DMD), a severe condition that ...
Apr 30, 2025
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Ndeye Lam visits the cemetery often, praying and gently touching the seashells laid out across her daughter's gravesite.
Apr 27, 2025
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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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