Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial - The Lancet

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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial - The Lancet

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PDF) Intracerebroventricular 2-hydroxypropyl-β-cyclodextrin improves not only neurological symptoms but also hepatic abnormalities in Niemann-Pick disease type C model mice and patients

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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial - The Lancet

Differential mode of cholesterol inclusion with 2‐hydroxypropyl‐cyclodextrins increases safety margin in treatment of Niemann‐Pick disease type C - Yamada - 2021 - British Journal of Pharmacology - Wiley Online Library

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