Researchers at Baylor College of Medicine have linked specific variants or mutations of the gene myocardin-related transcription factor B (MRTFB) with a novel neurodevelopmental disorder.
May 9, 2023
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Goldenhar syndrome is a rare congenital disease, affecting early fetal development. This syndrome includes malformations of varying severity, affecting different parts of the face. Its causes and modes of transmission are ...
May 4, 2023
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Researchers at Karolinska Institutet have mapped how cells in the human spinal cord are formed in the embryo and what genes control the process. Their findings can give rise to new knowledge on how injury to and diseases ...
Apr 26, 2023
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Premature infants, especially very low birthweight babies, are at risk for intraventricular hemorrhage. A frequent complication of these brain bleeds is hydrocephalus, an accumulation of cerebrospinal fluid (CSF) in the brain ...
Apr 24, 2023
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How is the mammals' heart formed? What role do endothelial cells play? Do the same pathways and genes involved in the formation of the embryonic heart also play a role in repair processes? A new study conducted by a team ...
Apr 3, 2023
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Providing an overview of the submissions process and examples of U.S. Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help ...
Mar 27, 2023
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Researchers at Texas A&M University have developed the first molecular therapeutic for Angelman syndrome to advance into clinical development.
Mar 23, 2023
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The use of a monoclonal antibody that reduced circulating IgG levels, led to a decrease in preexisting neutralizing antibodies (NAbs) to adeno-associated virus (AAV). The study, which showed that this strategy enabled gene ...
Mar 22, 2023
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National Institutes of Health researchers compared a new genetic animal model of Down syndrome to the standard model and found the updated version to be enhanced. The new mouse model shows milder cognitive traits compared ...
Mar 14, 2023
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Leukemia (blood cancer) is a group of malignant and aggressive diseases of the blood-forming cells in the bone marrow. Very intensive chemotherapy and in some cases a bone marrow transplant are the only cure. Like all cancers, ...
Mar 10, 2023
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