MeSH tree: C16.320.322
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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In a clinical practice guideline issued by the International Society on Thrombosis and Haemostasis and published online June 13 in the Journal of Thrombosis and Haemostasis, recommendations are presented for the treatment ...
Jul 1, 2024
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Studies of mouse models of Fragile X syndrome and Phelan-McDermid syndrome show that treatment with cannabidiol (CBD) and the diabetes drug metformin can alleviate behavioral difficulties, according to research presented ...
Jun 25, 2024
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The U.S. Food and Drug Administration has expanded the approval of Sarepta Therapeutics' Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in individuals ≥4 ...
Jun 25, 2024
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Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden's rare, inherited immune deficiency. But it's not available to him.
Jun 21, 2024
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The U.S. Food and Drug Administration has approved the first generic version of Emflaza (deflazacort) oral suspension for Duchenne muscular dystrophy (DMD). Approval of the generic version of Emflaza oral suspension was granted ...
Jun 17, 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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By studying mutations in yeast and human cells, Johns Hopkins Medicine scientists say they have found that biochemical bonds between fats and proteins in the mitochondrion, the cell's powerhouse, play a crucial role in how ...
Jun 7, 2024
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When the myelin sheath that surrounds nerve fibers in the brain and spinal cord becomes damaged, a number of debilitating conditions can result that limit mobility, inhibit independence and reduce life expectancy. Multiple ...
Jun 6, 2024
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Researchers from Bar-Ilan University, in collaboration with Sheba Medical Center, have developed a novel platform to model human muscle diseases in the C. elegans worm. This innovation facilitates the study of diseases in ...
May 15, 2024
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