Records containing: Niemann-Pick Disease, Type C
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CLINICAL_TRIALS study record update · 2026-04-20Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
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CLINICAL_TRIALS study record update · 2026-04-20Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
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CLINICAL_TRIALS study record update · 2026-04-03Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study
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CLINICAL_TRIALS study record update · 2025-12-12Phase 1/2a Study of 2-Hydroxypropyl-Beta-Cyclodextrin Therapy for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
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CLINICAL_TRIALS study record update · 2025-09-12ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
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CLINICAL_TRIALS study record update · 2025-03-30A Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat (Zavesca) for the Treatment of Niemann Pick Type C Disease (NPC) in Chinese Subjects
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CLINICAL_TRIALS study record update · 2025-03-20Randomized, Double Blind, Placebo Controlled, Multicenter, 12 Weeks Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease
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CLINICAL_TRIALS study record update · 2024-08-16Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US
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CLINICAL_TRIALS study record update · 2023-12-26A Phase 2B/3 Open-label Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 Disease Previously Treated Under Protocol VTS301
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CLINICAL_TRIALS study record update · 2023-11-28Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study.
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CLINICAL_TRIALS study record update · 2023-08-29A Phase 2B/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
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CLINICAL_TRIALS study record update · 2023-06-09Arimoclomol Prospective Double-blind, Randomised, Placebo-controlled Study in Patients Diagnosed With Niemann-Pick Disease Type C
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CLINICAL_TRIALS study record update · 2023-02-22A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled 3-Part Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
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CLINICAL_TRIALS study record update · 2023-02-13Biomarker for Niemann Pick Type C Disease (NPC1/NPC2) an International, Multicenter, Epidemiological Study
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CLINICAL_TRIALS study record update · 2021-06-07Complement Activation in the Lysosomal Storage Disorders
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CLINICAL_TRIALS study record update · 2020-07-22An Open-label, Multicenter Safety and Tolerability Study of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Pediatric Subjects Aged < 4 Years With Neurologic Manifestations of Niemann-Pick Type C (NPC) Disease
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CLINICAL_TRIALS study record update · 2017-05-18A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C in Order to Characterise the Individual Patient Disease Profile and Historic Signo-symptomatology Progression Pattern
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CLINICAL_TRIALS study record update · 2016-01-06Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
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CLINICAL_TRIALS study record update · 2012-07-12Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
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CLINICAL_TRIALS study record update · 2011-07-13Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplantation