Researchers at the University of Notre Dame have targeted NPC since 2003. With partnerships through the Boler-Parseghian Center for Rare and Neglected Diseases, there have been multiple studies funded to identify the molecular mechanisms behind NPC disease, develop new NPC disease models, identify novel techniques to better understand the disease, and support new young investigators to perform research on NPC disease.

As a result of the initial partnership in 2010, Notre Dame researchers established the William K. Warren Center for Drug Discovery and Development, a resource any member of the worldwide NPC research community may utilize for the design and synthesis of potential drug candidates. The Center enjoys multidisciplinary collaborations with Cornell, Columbia, Tufts, Washington University, and Texas Southwestern medical centers along with researchers at Purdue and Scripps Research Institute. Compounds produced in the Center are now under active investigation in all of these collaborators’ labs.

Several Notre Dame faculty are involved in NPC research including Kevin Vaughan, Paul Helquist, Olaf Wiest, Brian Blagg, Rich Taylor, Chris Patzke, and Matt Champion. Their work covers a wide range of approaches, computational and synthetic strategies in chemistry, cellular pathways, protein function and detection, engineering, clinical outreach as well as, induced pluripotent stem cells for NPC therapies.