Cerebrospinal Fluid Calbindin D Concentration as a Biomarker of Cerebellar Disease Progression in Niemann-Pick Type C1 Disease.

@article{Bradbury2016CerebrospinalFC,
  title={Cerebrospinal Fluid Calbindin D Concentration as a Biomarker of Cerebellar Disease Progression in Niemann-Pick Type C1 Disease.},
  author={Allison M. Bradbury and Jessica H. Bagel and Maureen L. Sampson and Nicole Yanjanin Farhat and Wenge Ding and Gary P Swain and Maria L Prociuk and Patricia O'donnell and Kenneth J Drobatz and Brittney L. Gurda and Christopher A Wassif and Alan T Remaley and Forbes D Porter and Charles Herman Vite},
  journal={The Journal of pharmacology and experimental therapeutics},
  year={2016},
  volume={358 2},
  pages={254-61}
}
Niemann-Pick type C (NPC) 1 disease is a rare, inherited, neurodegenerative disease. Clear evidence of the therapeutic efficacy of 2-hydroxypropyl-β-cyclodextrin (HPβCD) in animal models resulted in the initiation of a phase I/IIa clinical trial in 2013 and a phase IIb/III trial in 2015. With clinical trials ongoing, validation of a biomarker to track disease progression and serve as a supporting outcome measure of therapeutic efficacy has become compulsory. In this study, we evaluated calcium… CONTINUE READING
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