Novel alpha-glucosaminidase for Sanfilippo C syndrome
Awardee: Patricia Dickson
Institution: Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
Award Amount: $59,449
Funding Period: January 1, 2018 - December 31, 2018
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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
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