Daniel Ory, MD

2012 SPIRiT Pilot awardee, Daniel Ory, MD, moved a newly approved FDA-therapy to Phase 2b/3 clinical trials for Niemann-Pick type C1 (NPC1) disease. The research was featured in an NCATS Information release highlighting the Therapeutics for Rare and Neglected Diseases (TRND) Program and a recent paper describing the process for fostering collaborative research for rare genetic diseases modeling the NPC1 experience.