A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-β-cyclodextrin for acute liver disease in infants with Niemann-Pick C1

Keyphrases

• Liver Disease 100%
• Niemann-Pick C1-like 1 (NPC1L1) 100%
• Cholesterol 100%
• Hydroxypropyl-β-cyclodextrin (HP-β-CD) 100%
• Niemann-Pick C 100%
• Non-randomized Clinical Trial 100%
• Acute Liver Disease 100%
• Liver 50%
• Glycine 50%
• Autosomal Recessive 50%
• Central Nervous System 50%
• Lysosome 50%
• Endolysosomal 50%
• Lysosomal Storage Disease 50%
• Bile Acids 50%
• Unesterified Cholesterol 50%
• NPC2 50%
• NPC1 Protein 50%
• Primary Outcome Measure 50%
• Self-limiting 50%
• Drug Adverse Events 50%
• Binding Agent 50%
• Drug Studies 50%
• Newborn Period 50%
• NPC2 Gene 50%
• Liver Enzymes 50%
• Investigational New Drug 50%
• Open-label Clinical Trial 50%
• Direct Hyperbilirubinemia 50%
• Cholesterol Binding 50%

Medicine and Dentistry

• Clinical Trial 100%
• Liver Disease 100%
• Cyclodextrin 100%
• Adverse Event 25%
• Infusion 25%
• Autosomal Recessive Disorder 25%
• Newborn Period 25%
• Lysosome 25%
• Lysosomal Storage Disease 25%
• Central Nervous System 25%
• Bile Acid 25%
• Liver Enzyme 25%
• Hyperbilirubinemia 25%

Pharmacology, Toxicology and Pharmaceutical Science

• Clinical Trial 100%
• Liver Disease 100%
• Cyclodextrin 100%
• Adverse Event 25%
• Lysosome Storage Disease 25%
• Bile Acid 25%
• Autosomal Recessive Disorder 25%
• Hyperbilirubinemia 25%
• Liver Enzyme 25%