Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
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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 has passed its completion date and status has not been verified in more than two years.