Cambridge, Mass. – By analyzing millions of small genetic differences across a person’s genome, researchers can calculate a polygenic risk score to estimate someone’s lifetime odds of developing a certain disease. Over the past decade, scientists have developed these risk scores for dozens of diseases, including heart disease, kidney disease,...
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Altering a protein linked to Rett syndrome so that it cannot bind to a particular type of DNA tag results in traits reminiscent of the syndrome, according to a new study in mice. The findings could help explain the syndrome’s delayed onset in people: Because the tag does not arise until after...
Boston, Mass. — Childhood interstitial lung disease (chILD) is a group of rare, genetic lung disorders affecting infants and children who typically present with a range of symptoms, such as shortness of breath, rapid breathing and coughing. While many causal genes have been identified for chILD, understanding its pathogenesis has...
Novato, Calif. – Restricting calories is known to improve health and increase lifespan, but much of how it does so remains a mystery, especially in regard to how it protects the brain. Buck scientists have uncovered a role for a gene called OXR1 that is necessary for the lifespan extension...
ITHACA, N.Y. — By studying individuals who spontaneously clear hepatitis C infections, a team of researchers has identified viable vaccine targets for a disease that infects 70 million worldwide with case numbers increasing every year. It turns out that a quarter of people who become infected with the hepatitis C virus...
Coronary artery bypass surgery provides long-term benefits for children whose hearts and blood vessels are damaged by Kawasaki disease, Japanese researchers report in Circulation: Journal of the American Heart Association. Researchers followed 114 people for up to 25 years who had bypass surgery as children or adolescents (ages 1 to...
Tucson, Arizona — Critical Path Institute’s (C-Path) Duchenne Regulatory Science Consortium (D-RSC) is excited to announce the launch of a groundbreaking model-based Clinical Trial Simulator (CTS), specifically designed to improve design of efficacy studies for potential therapies for Duchenne muscular dystrophy (DMD). This pioneering Drug Development Tool is set to...
Tucson, Arizona — Critical Path Institute (C-Path) today announced the formation of a new task force under its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®), dedicated to advancing therapeutic development for Progressive Supranuclear Palsy (PSP). This initiative brings together leading organizations and experts in a concerted effort to tackle...
Tucson, AZ. – Critical Path Institute (C-Path) is pleased to announce the release of a new peer-reviewed publication, titled “Transforming Drug Development for Neurological Disorders: Proceedings from a Multi-disease Area Workshop,” now published in Neurotherapeutics, The Journal of the American Society for Experimental Neurotherapeutics. A distinguished team of C-Path scientists...
Rochester, NY – New research has identified the specific biological mechanism behind the muscle dysfunction found in myotonic dystrophy type 1 (DM1) and further shows that calcium channel blockers can reverse these symptoms in animal models of the disease. The researchers believe this class of drugs, widely used to treat...