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The latest rare diseases news from News Medical |
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 | | |  Vitamin B3 shows promise for treating rare genetic disorder Scientists at Gladstone Institutes have flipped the traditional approach to finding potential treatments for deadly diseases. | | | | |  Could a rare gene variant help reduce smoking? New study suggests it might An exome-wide association study of nearly 38,000 smokers from the Mexico City Prospective Study identified rare coding variants in CHRNB3 that are associated with substantially fewer cigarettes smoked per day. Cross-ancestry analyses in European and East Asian datasets support a role for the β3 nicotinic receptor subunit in modulating smoking intensity. | | | | |  MSK uncovers how interacting mutations shield breast cancer Researchers at Memorial Sloan Kettering Cancer Center (MSK) have made an important discovery about how genetic mutations in breast cancer patients can interact and drive resistance to certain drugs called CDK4/6 inhibitors. This finding, published in Nature, suggests a new strategy for predicting and preventing resistance to specific therapies based on the tumor's genetic profile. | | | | |  Global health governance? Power shifts in the era of AI The dynamics of global health governance are shifting with AI advancements, impacting drug discovery, equity, and the role of multilateral institutions. | |
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| | | | |  Researchers at the Cancer Research Institute and the Nano Life Science Institute (WPI-NanoLSI), Kanazawa University, have uncovered a critical mechanism that enables gastric cancer to spread to distant organs. | | | | |  A team of researchers at Texas Children's Duncan Neurological Research Institute (NRI) and Baylor College of Medicine report in Science Translational Medicine a potential new approach to treat Rett syndrome – offering early promise for a rare neurodevelopmental disorder that currently has no cure. | | | | |  MIT researchers have discovered that two common genetic mutations that cause Rett syndrome each set off a molecular chain of events that compromises the structural integrity of developing brain blood vessels, making them leaky. | | | | | Gibson Oncology, a clinical-stage private pharmaceutical company headquartered in Miami, announced it has entered Phase 2 clinical trials with LMP744 for the treatment of first-time recurrent glioblastoma patients. | | | | | A tiny region in a little-known muscle protein may hold the key to a healthy, steady heartbeat, as well as possible clues to future treatment of devastating heart ailments. | | | | | The new generation of Alzheimer's disease drugs - the first proven to change the course of the disease - typically extend independent living for patients by 10 months. | |
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