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  • Author(s):
    Md Suhail Alam, Bruce Cooper, Kasturi Halder
    Abstract:

    Histone deacetylase (HDAC) inhibitors are of significant interest as drugs. However, their use in neurological disorders has raised concern because HDACs are required for brain function. We have previously shown that a triple combination formulation (TCF) of the pan HDACi vorinostat (Vo) improves pharmacokinetic exposure and entry of Vo into the brain. TCF treatment significantly delayed both neurodegeneration and death in the Npc1nmf164 murine model of Niemann Pick Type C (NPC) disease. The …

    Date Published:
    2017-09
    Record Visibility:
    Public
  • 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