A humanised Drosophila melanogaster model of MPSIIIb to rapidly study central nervous system efficacy of combined chaperone and substrate reduction therapy

2017MPS
Written By Million Dollar Bike Ride

Awardee: Emyr Lloyd-Evans

Institution: Cardiff University

Award Amount: $59,449

Funding Period: January 1, 2018 - December 31, 2018

Million Dollar Bike Ride
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Identification and characterization of novel cell-permeable, small molecule adenylyl cyclase inhibitors for future development as drugs to treat FD/MAS
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Novel alpha-glucosaminidase for Sanfilippo C syndrome