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  Article

  Pluronic based β-cyclodextrin polyrotaxanes for treatment of Niemann-Pick Type C disease
  

  Author(s):

  Christopher J. Collins, Bradley P. Loren, Md Suhail Alam, Yawo Mondjinou, Joseph L. Skulsky, Cheyenne R. Chaplain, Kasturi Haldar, David H. Thompson

  Abstract:

  Niemann-Pick Type C disease (NPC) is a rare metabolic disorder characterized by disruption of normal cholesterol trafficking within the cells of the body. There are no FDA approved treatments available for NPC patients. Recently, the cycloheptaglucoside 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) has shown efficacy as a potential NPC therapeutic by extending lifetime in NPC mice, delaying neurodegeneration, and decreasing visceral and neurological cholesterol burden. Although promising, systemic…

  Date Published:

  2017

  Record Visibility:

  Public