A study of the correlation between gait abnormalities, activity monitoring parameters, CMTPeds and a biomarker in children with Charcot-Marie-Tooth disease

MDBRCMT
Written By Million Dollar Bike Ride

Awardee: Sylvia Ounpuu

Institution: Connecticut Children's Medical Center

Award Amount: $60,330

Million Dollar Bike Ride
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A novel approach for identification of therapeutic leads to the Maple Syrup Urine Disease
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Characterisation of CRB1 patient human iPSC-derived retinal organoids to set-up potency assays for gene therapy