Intrathecal 2-hydroxypropyl-�-cyclodextrin Decreases Neurological Disease Progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial
Lancet 390:1758-1768, Ory, D.S.,et al, 2017
Clinicopathologic Conference, Cerebral Infarction from Internal Carotid Occlusion Related to Sickle Cell Arteriopathy
NEJM 374:1265-1275, Case 10-2016, 2016