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Currently, there are no FDA-approved therapies for Niemann-Pick disease type-C1 (NPC). NPC is a rare lethal genetic lysosomal storage disorder that results in an accumulation of cholesterol in the liver and spleen and eventually leads to neurodegeneration. 2-hydroxypropyl-β-cyclodextrin (HPβCD) is a cyclodextrin typically used by the pharmaceutical industry as an excipient. Studies of NPC in animal models have shown that HPβCD can reduce the biochemical burden associated with NPC, improving neurological pathology, decreasing neurological dysfunction, and increasing lifespan. 

Diffusion tensor imaging (DTI) is an MRI method that produces in vivo magnetic resonance images of biological tissues sensitized with the localized and contrasting characteristics of water diffusion, producing microscopic images of tissues. Water molecules become excited when exposed to a strong magnetic field, which causes the protons in water molecules to move in a coordinated and precise way. The intensity of each image element (voxel) reflects the best estimate of the rate of water diffusion at that location.