A recent large cohort study, conducted jointly by researchers from BGI Genomics and Nanjing Women and Children's Healthcare Hospital, implemented a self-developed prenatal screening strategy to effectively identify and classify ...
Jul 17, 2024
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Hepatic, or liver, disease affects more than 100 million people in the U.S. About 4.5 million adults (1.8%) have been diagnosed with liver disease, but it is estimated that between 80 and 100 million adults in the U.S. have ...
Jul 17, 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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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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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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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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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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Experts are working on novel immune-enhancing therapies called host-directed therapies to use the body's own immune system to target tuberculosis, with hopes that they could tackle even the drug-resistant forms of the disease. ...
Apr 26, 2024
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The U.S. Food and Drug Administration has approved Duvyzat (givinostat) as an oral medication for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older.
Mar 26, 2024
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