NIH scientists pursue new therapies to improve rare disease drug development

NIH: September 12, 2013

Four new pre-clinical drug development projects at the National Institutes of Health will target a form of blindness and diseases characterized by cardiac problems. The projects were selected for their potential to treat specific rare diseases and to help scientists uncover new information that can be shared with other researchers.

The studies will be funded through the Therapeutics for Rare and Neglected Diseases (TRND) program at the NIH’s National Center for Advancing Translational Sciences (NCATS). This group of projects also marks the TRND program’s first use of stem cells as well as its first collaboration with a large pharmaceutical company, Eli Lilly, to co-develop a treatment for a rare disease. Read More