TrialLancet (London, England)2017

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

Daniel S Ory et al.PubMed ↗Full text ↗Publisher ↗

No numbers read from the abstract.