Identification and characterization of novel cell-permeable, small molecule adenylyl cyclase inhibitors for future development as drugs to treat FD/MAS

MDBRFDMAS
Written By Million Dollar Bike Ride

Awardee: Charles Hoffman

Institution: Boston College

Award Amount: $68,185

Funding Period: February 1, 2019 - January 31, 2020

Million Dollar Bike Ride
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Single cell transcriptome analysis of skeletal stem cells derived from FD/MAS patients
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A humanised Drosophila melanogaster model of MPSIIIb to rapidly study central nervous system efficacy of combined chaperone and substrate reduction therapy