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Intrathecal 2-hydroxypropyl-�-cyclodextrin Decreases Neurological Disease Progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial
Lancet 390:1758-1768, Ory, D.S.,et al, 2017
See this aricle in Pubmed
Article Abstract
Patients with NPC1 treated with intrathecal HPBCD had slowed disease progression with an acceptable safety profile. These date support the initiation of a multinational randomized, controlled trial of intrathecal HPBCD.
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