Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
|2||Not yet recruiting||
Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
|3||Not yet recruiting||
Study of Pharmacokinetics and Preliminary Efficacy in Patients With Niemann-Pick C1
† Study has passed its completion date and status has not been verified in more than two years.