Our scientists have been rewarded a grant for further research…. we’re getting closer and closer as each dollar gets committed to NPCD research.
Million Dollar Bike Ride Grant Program from Orphan Disease Center, University of Pennsylvania
Principal Investigator: Professor Ashley Bush
Project title: Dual targeting of defective lipid and metal pathways in Niemann-Pick type C disease: an in vitro pilot study
The project proposes to undertake a pilot study to screen and identify potent combinations of metal-modulating compounds and miglustat and/or cyclodextrin that will restore cellular metal and cholesterol balance using NP-C1 patient fibroblast cells harbouring different NPC1 mutations. The long-term goal is to develop a synergistic combination therapy that will prolong the life of NP-C patients and restore their neurological and other functions.
Award amount: US$50,000
Award period: 1/1/2015 – 31/12/2015