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

Awardee: Tommaso Pizzorusso

Institution: University of Florence, Department NEUROFARBA

Award Amount: $51,382

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

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

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Anti-resorptive drugs in Fibrous Dysplasia of bone: studies on the effects of a RANKL inhibitor and Zoledronic Acid in a murine model of the disease by radiography, histology