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1. Intrathecal 2-Hydroxypropyl-Beta-Cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick Disease Type C (NPC).

Author: Sakiyama, Y., Shibata, S., Sanayama, H., Ono, S., Maekawa, M., Matsuo, M., Irie, T., and Eto, Y.
Subjects: *CYCLODEXTRINS, *DRUG therapy, *NIEMANN-Pick diseases, *MEDICAL research, DISEASES in adults
Published: 2015
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