Pre-clinical animal model and cell evaluation of candidate therapies for human NUBPL disease. MDBRNUBPL Feb 1 Written by Million Dollar Bike Ride Awardee: Marni FalkInstitution: Children's Hospital of Philadelphia and University of PennsylvaniaAward Amount: $61,134 MDBRNUBPL2019 Million Dollar Bike Ride Previous Previous Development of novel human stem cell models of Beta-Propeller Protein- Associated Neurodegeneration (BPAN) for disease modelling and drug screening Next Next Genetic Mechanisms in Neuroendocrine cell Hyperplasia of Infancy (NEHI)
Pre-clinical animal model and cell evaluation of candidate therapies for human NUBPL disease. MDBRNUBPL Feb 1 Written by Million Dollar Bike Ride Awardee: Marni FalkInstitution: Children's Hospital of Philadelphia and University of PennsylvaniaAward Amount: $61,134 MDBRNUBPL2019 Million Dollar Bike Ride