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.
Cristan A FarmerAudrey ThurmNicole FarhatSimona BianconiLee Ann KeenerForbes D PorterPublished in: CNS drugs (2020)
ClinicalTrials.gov identifier NCT01747135: Hydroxypropyl Beta Cyclodextrin for Niemann-Pick type C1 Disease.