2-hydroxypropyl- & #946;-cyclodextrins and blood-brain barrier in Niemann-Pick Disease type C1

(Bentham Science Publishers) The rare, chronic, autosomal-recessive lysosomal storage disease Niemann-Pick disease type C1 (NPC1) is characterized by progressively debilitating and ultimately fatal neurological manifestations. There is an urgent need for disease-modifying therapies that address NPC1 neurological pathophysiology; and passage through the blood-brain barrier represents an important consideration for novel NPC1 drugs.
Source: EurekAlert! - Biology - Category: Biology Source Type: news