News

Vienna to Host RARE2019 Meeting on Rare Diseases

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…

Fast Screening Strategy Identifies Potential New Inhibitors for Huntington’s Disease

Several small molecules have been identified by the Alborada Drug Discovery Institute as potential inhibitors to help halt the underlying mechanisms that drive the development of neurodegenerative diseases such as Huntington’s and Parkinson’s disease. Silencing phosphatidylinositol 5-phosphate 4 (PI5P4) kinases has been shown to enhance the natural cleaning system of cells, called autophagy,…

Phase 3 Trial of RG6042 Will Gauge Potential of Gene-silencing Approaches for Huntington’s, Other Diseases

The upcoming Phase 3 trial of RG6042 will test whether lowering the levels of a mutant form of huntingtin (mHTT) translates into functional, motor and cognitive benefits for people with Huntington’s disease. Potential benefits could also help advance gene-silencing approaches in other neurodegenerative diseases. “Deep down, we all…

Brain Neurons Disconnect from Each Other in Process Called Neuritosis, Mouse Study of Huntington’s Reveals

The progressive death of brain nerve cells observed in Huntington’s disease is likely preceded by the retraction of their neurites — long projections that establish connections throughout the brain, a mouse study reveals. Understanding this mechanism, which is exacerbated during aging, stress, and neurodegenerative diseases, may open new avenues…