News

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…

Experimental immunotherapy laquinimod reduced brain atrophy in the caudate nucleus, one of the most affected brain areas in Huntington’s disease, and other cerebral regions but it did not improve motor function in recently diagnosed patients, LEGATO-HD study findings show. Results from the trial were recently discussed at the…

DYST 201, a gene therapy targeting the mutated forms of the gene responsible for Huntington’s disease, has shown promising results, Dystrogen Therapeutics announced. The results will be presented at two meetings in Krakow, Poland — the July 6-11 44th FEBS Congress and the June 11-16 24th Annual Meeting of the RNA Society.