Small molecule modulators of telomerase as novel treatments for dyskeratosis congenita

Awardee: Suneet Agarwal

Institution: Boston Children's Hospital

Award Amount: $100,373

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

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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

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The use of a patient-centric neuronal cell model of Beta-Propeller Protein-Associated Neurodegeneration (BPAN) as a platform to develop novel therapies