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Clinical Trials for Niemann-Pick Diseases

• 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
• A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
• Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
• Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
• Safety and Efficacy of Miglustat in Chinese NPC Patients
• Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C
• Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
• 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)
• Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease
• Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
• Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
• Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease
• Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
• Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
• Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C
• VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
• Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
• Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1
• 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
• Application of Miglustat in Patients With Niemann-Pick Type C
• Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
• Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients
• Human Placental-Derived Stem Cell Transplantation
• Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
• Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
• Miglustat in Niemann-Pick Type C Disease
• Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
• Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease
• Stem Cell Transplant for Inborn Errors of Metabolism
• PET Scan of Brain Metabolism in Relation to Age and Disease