Preclinical studies of FDA-approved small-molecules implementing lysosomal activity in a mouse model of MPSIIIA

MDBRMPS
Written By Million Dollar Bike Ride

Awardee: Diego Luis Medina

Institution: Telethon Institute of Genetics and Medicine (TIGEM)

Award Amount: $$55,992

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

Million Dollar Bike Ride
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Expanding the Breadth of Metabolite Analysis for Snyder Robinson Syndrome
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Generation of lysosomal enzyme variants with enhanced therapeutic potential for the treatment of MPS