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
Intrathecal Administration of Autologous Mesenchymal Stem Cells in Multiple System Atrophy
Neurol 93:e77-e87, Singer, W.,et al, 2019