CAS : 94035-02-6
Molecular formula
: C54H102O39
Molecular Weight :
1375.36
Hydroxypropyl beta cyclodextrin (HPBCD) is being tested for a disease called Niemann-Pick disease
type C1 (NPC1). NPC1 is a genetic disorder that results in gradual loss of
nervous system function. Cholesterol and other fats have trouble moving out of
the brain cells, which makes the cells work poorly and leads to symptoms. There
is no treatment currently approved in the US for NPC1. Researchers want to
test if it is safe to use HPBCD for NPC1. They want to see if it can help brain
cells process cholesterol better.