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

ABSTRACT:

SUBMITTER: Ory DS 

PROVIDER: S-EPMC6176479 | biostudies-literature | 2017 Oct

REPOSITORIES: biostudies-literature