A multi-center, prospective, minimal intensity BMT trial for dyskeratosis congenita

2016DC
Written By Million Dollar Bike Ride

Awardee: Agarwal Suneet

Institution: Boston Children's Hospital

Award Amount: $51,000

Funding Period: January 1, 2017 - December 31, 2017

Million Dollar Bike Ride
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Treatment of adult polyglucosan body disease caused by an intronic mutation in glycogen branching enzyme gene.
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Impact on Niemann-Pick C disease of the cross-talk between the c-Abl and the TFEB signaling pathways