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

MDBRCMT

Feb 1

Written By Million Dollar Bike Ride

Awardee: Sylvia Ounpuu

Institution: Connecticut Children's Medical Center

Award Amount: $60,330

MDBRCMT2019

Million Dollar Bike Ride

A novel approach for identification of therapeutic leads to the Maple Syrup Urine Disease

Characterisation of CRB1 patient human iPSC-derived retinal organoids to set-up potency assays for gene therapy