MeSH tree: C16.320.565
In a new study, Broad Institute researchers report that two small-molecule chemicals that reduce oxygen delivery to cells improved symptoms of neurodegeneration in a mouse model of Parkinson's disease. The scientists had ...
Oct 12, 2026
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Biomedical engineers at Duke University have developed a three-layer model of human blood vessels that enables researchers to better understand how Hutchinson-Gilford progeria syndrome (HGPS), a rare genetic disorder that ...
Oct 9, 2026
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In hypophosphatasia, a rare inherited condition that weakens bones and teeth, early tooth loss can be the first or even the only obvious sign. Yet why dental symptoms differ so much between patients has remained unclear.
Oct 6, 2026
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Researchers at the Greenwood Genetic Center (GGC) led the discovery of an unexpected connection between inherited blindness and a gene known for causing a rare metabolic disorder.
Oct 6, 2026
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A new report has revealed critical gaps in the care of Australian children with a preventable pediatric disorder that can lead to premature cardiovascular disease (CVD). Familial hypercholesterolemia (FH) is a common genetic ...
Sep 30, 2026
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The Abigail Wexner Research Institute at Nationwide Children's Hospital celebrated the Food and Drug Administration's (FDA) approval of FAYUVI for Sanfilippo syndrome type A, following years of research in its Jerry R. Mendell ...
Sep 23, 2026
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The Food and Drug Administration recently approved the first drug to treat Alexander disease, a rare, progressive and often fatal neurological disorder that can affect motor, cognitive and autonomic functions like heart rate ...
Sep 21, 2026
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Our cells have their own internal "garbage collection" system that gathers up everything that is defective, worn out or foreign. Acting as a sort of "cellular stomach," the so-called lysosomes are responsible for this recycling ...
Sep 15, 2026
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Most of us are used to hearing about the risks of not getting enough iron. Yet having too much iron in the body can also cause serious health problems.
Sep 15, 2026
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Researchers have taken an important step toward solving one of the biggest challenges in gene therapy for children with metabolic liver disorders: growth itself. As a child's liver grows, some gene therapies that once worked ...
Sep 11, 2026
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