Main Menu

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.

Niemann-Pick disease, type C (NPC) is a lethal, neurodegenerative disorder that affects children. Presently, no therapies for NPC are approved by the Food and Drug Administration (FDA). Several studies have suggested the potential use of 2-hydroxypropyl-β-cyclodextrin (HPBCD) to treat NPC, but the critical studies and data required for an Investigative New Drug (IND) application to evaluate HPBCD were not available.