Primary Recipient

MOUNT SINAI SCHOOL OF MEDICINE

Amount

$466,125.00

Award Date

5/15/2009

Administering Agency

Health and Human Services, Department of / National Institutes of Health

CDFA Number

93.701

CDFA Program Title

Trans-NIH Recovery Act Research Support

Description

NPC1 disease is a severe lysosomal lipidosis in which the egress of cholesterol and other lipids from the endosomal/lysosomal (E/L) system is impeded, leading to severe neurodegeneration and premature demise. At present there are no treatment options for