AAVrh.10-mediated Npc2 Gene transfer for the treatment of Npc2 Deficient Niemann-Pick disease

2017NPC

Jan 1

Written By Million Dollar Bike Ride

Awardee: Dolan Sondhi

Institution: Weill Cornell Medicine

Award Amount: $49,645

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

MDBR2017NPC

Million Dollar Bike Ride

Epigenetic Therapy to Treat Pitt-Hopkins Syndrome (PTHS)

Targeting molecular chaperone HSP90 to treat NPC1 disease