14 studies found for: Open Studies | "Niemann-Pick Diseases"
Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C
Biomarker for Niemann Pick Type C Disease
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
Study of Pharmacokinetics and Preliminary Efficacy in Patients With Niemann-Pick C1
Study of VTS-270 (2-hydroxypropyl-β-cyclodextrin) to Treat Niemann-Pick Type C1 (NPC1) Disease
Investigating Lysosomal Storage Diseases in Minority Groups
Human Placental-Derived Stem Cell Transplantation
Genetic Characterization of Movement Disorders and Dementias
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Molecular and Cellular Mechanisms of Lysosomal Storage Diseases
† Study has passed its completion date and status has not been verified in more than two years.