Adeno-associated virus serotype 9-mediated gene therapy for Adrenomyeloneuropathy

2014ALD

Jan 1

Written By Million Dollar Bike Ride

Awardee: Florian Eichler

Institution: Massachusetts General Hospital / Harvard Medical School

Award Amount: $50,000

Funding Period: January 1, 2015 - December 31, 2015

MDBR2014ALD

Million Dollar Bike Ride

CNS gene therapy to restore NPC1 protein expression in cerebellar Purkinje cells and delay or prevent disease onset in feline Niemann-Pick type C1 (NPC1) disease.

Pilot Study of the Efficacy and Safety of Sirolimus in the Treatment of Congenital Hyperinsulinism