Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-β-Cyclodextrin

Published 2018 View Full Article

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Title
Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-β-Cyclodextrin
Authors
Keywords
Niemann-Pick type C, Lysosomal storage disorder, Neurodegenerative disease, Cholesterol, Cyclodextrin
Journal
PEDIATRIC NEUROLOGY
Volume 80, Issue -, Pages 24-34
Publisher
Elsevier BV
Online
2018-01-09
DOI
10.1016/j.pediatrneurol.2017.12.014

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