Exploring the therapeutic potential of RANKL inhibition in Fibrous Dysplasia of bone: studies on murine transgenic models of the disease.

Awardee: Mara Riminucci

Institution: Sapienza University of Rome

Award Amount: $53,614

Funding Period: January 1, 2018 - December 31, 2018

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Targeting molecular chaperone HSP90 to treat NPC1 disease

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Mechanistic study of WDR45 in the autophagy pathway and neurodegeneration