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

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 clinic...

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Pubblicato in:J Pharmacol Exp Ther
Autori principali: Bradbury, Allison, Bagel, Jessica, Sampson, Maureen, Farhat, Nicole, Ding, Wenge, Swain, Gary, Prociuk, Maria, O’Donnell, Patricia, Drobatz, Kenneth, Gurda, Brittney, Wassif, Christopher, Remaley, Alan, Porter, Forbes, Vite, Charles
Natura: Artigo
Lingua:Inglês
Pubblicazione: The American Society for Pharmacology and Experimental Therapeutics 2016
Soggetti:
Drug Discovery and Translational Medicine
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4959104/
https://ncbi.nlm.nih.gov/pubmed/27307499
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1124/jpet.116.232975
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