NPC-1 is a rare, progressive and ultimately lethal genetic disorder affecting an estimated 2,000-3,000 patients globally. The results were published today in an article entitled, “Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1,” in PLOS ONE, the world’s first multidisciplinary, Open Access journal, and are available at http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0175478.
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