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 […]
Fumihiko Urano, MD, PhD
Leveraging 2011 Pilot funding and a 2016 JIT award to use services from the Regulatory Support Center (RSC), Fumihiko Urano, MD, PhD, secured IND approval for the use of dantrolene sodium in treatment of Wolfram Syndrome treatment. The first patients were enrolled in the Dantrolene Clinical Trial — NCT02829268 in Feb 2017. Learn more about […]