A comparative and quantitative approach to study lysosomal dysfunction in Mucopolysaccharidoses diseases

MDBRMPS
Written By Million Dollar Bike Ride

Awardee: Andrea Ballabio

Institution: Fondazione Telethon, TIGEM

Awarded Amount: $64,063

Million Dollar Bike Ride
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A Prospective Natural History Study of Mucolipidosis type 4
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Can we improve neurologic and psychiatric outcomes in MSUD? Studies in a new mouse model