Niemann-Pick Disease, Type C

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Safety and Efficacy of Miglustat in Chinese NPC Patients

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease

Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

Early Access Program With Arimoclomol in US Patients With NPC

Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

Biomarker for Niemann Pick Type C Disease (BioNPC)

Complement Activation in the Lysosomal Storage Disorders

Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C

Longitudinal Study of Cognition With Niemann-Pick Disease, Type C

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine