Long-Term Neuropsychological Outcomes from an Open-Label Phase I/IIa Trial of 2-Hydroxypropyl- β-Cyclodextrins (VTS-270) in Niemann-Pick Disease, Type C1

ConclusionIn conjunction with previous findings, these results provide support for the slowing of disease progress up to 36  months post-initiation of intrathecal VTS-270.RegistrationClinicalTrials.gov identifier NCT01747135: Hydroxypropyl Beta Cyclodextrin for Niemann-Pick type C1 Disease.
Source: CNS Drugs - Category: Neurology Source Type: research