A novel RNA-based CRISPR strategy for treating congenital muscular dystrophy

MDBRCMD

Feb 1

Written By Million Dollar Bike Ride

Awardee: Jamie Fitzgerald

Institution: Henry Ford Health System

Award Amount: $50,699

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

MDBRCMD2018

Million Dollar Bike Ride

Could exon skipping strategies be used as a treatment for recessive Ullrich Congenital Muscular Dystrophy?

Validation of pupil size as a biomarker for CDKL5 disorder: longitudinal assessment and relationship with disease severity