Development of novel human stem cell models of Beta-Propeller Protein- Associated Neurodegeneration (BPAN) for disease modelling and drug screening

MDBRNBIA(BPAN)NBIABPAN

Feb 1

Written By Million Dollar Bike Ride

Awardee: Paul Lockhart

Institution: Murdoch Children's Research Institute

Award Amount: $61,245

MDBRNBIABPANNBIABPAN2019

Million Dollar Bike Ride

Can we improve neurologic and psychiatric outcomes in MSUD? Studies in a new mouse model

Pre-clinical animal model and cell evaluation of candidate therapies for human NUBPL disease.