Genetic Cause of Als and Dementia Repaired by RNA-Targeting Strategy Developed at UF Scripps
Scientists at UF Scripps Biomedical Research have developed a potential medicine for a leading cause of ALS and dementia that works by eliminating disease-causing segments of RNA. The compound restored the health of neurons in the lab and rescued mice with the disease.
New Trial Drug Combination Shows Promise for Slowing Progression of ALS
A new drug combination tested in a multisite human clinical trial shows promise for slowing the progression of ALS, often called Lou Gehrig’s disease, researchers at the University of Florida and partner institutions reported in The New England Journal of Medicine. The oral drug, a combination of sodium phenylbutyrate and taurursodiol, was tested in the […]
New Study Shows Common Diabetes Drug Improves Symptoms in Genetic Form of ALS in Mice
University of Florida neuroscientists, Laura Ranum, Ph.D., and Tao Zu, Ph.D., showed in a mouse-model study that metformin, a widely prescribed drug approved by the U.S. Food and Drug Administration to treat Type 2 diabetes, reduces levels of specific mutant proteins central to the most common genetic form of ALS and frontotemporal dementia, or FTD, […]