Testing effectiveness (Phase 2)Ended earlyNCT03887533
What this trial is testing
Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
Who this might be right for
Niemann-Pick Disease, Type C1
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 2