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

MDBRMPS

Feb 1

Written By Million Dollar Bike Ride

Awardee: Andrea Ballabio

Institution: Fondazione Telethon, TIGEM

Awarded Amount: $64,063

MDBRMPS2019

Million Dollar Bike Ride

A Prospective Natural History Study of Mucolipidosis type 4

Can we improve neurologic and psychiatric outcomes in MSUD? Studies in a new mouse model