Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial
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| Description | scientific article published on 10 August 2017 |
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author: Cristin Davidson Rohini Sidhu Christopher P Austin William J Pavan |
| Publication date | August 10, 2017 |
| Language | English |
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