MeSH tree: C18.452.648
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 ...
16 hours ago
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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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An Australian research collaboration has identified several existing TGA-approved medicines that could help slow brain damage in children with a rare form of dementia—offering hope for faster treatment options.
Aug 20, 2026
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Much of how genetic risk leads to disease has remained hidden. In many cases, genetic variants linked to disease do not act on nearby genes along the chromosome. Instead, they influence genes located far away along the DNA ...
Aug 15, 2026
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I teach a university course called Hormones and Behaviour. What surprises students is that biological sex is not produced by a single switch. What surprises me is that many students have never been taught this before. It ...
Aug 14, 2026
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Researchers at the University of California San Diego and their colleagues have identified a key cellular pathway that drives brain degeneration in both a rare childhood disorder and the far more common Alzheimer's disease. ...
Aug 11, 2026
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A new study co-led by professor Valeriya Malysheva, group leader at the VIB-UAntwerp Center for Molecular Neurology, published in Nature Genetics, uncovers how the three-dimensional organization of DNA in rare immune cells ...
Aug 4, 2026
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Teeth and bones are among the hardest structures in the human body, but certain inherited conditions can rob them of that strength. One of them is hypophosphatasia (HPP), a rare, lifelong genetic bone disorder that disrupts ...
