{"id":"W3171494824","doi":"10.1016/j.ymgmr.2021.100772","title":"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","year":2021,"lang":"en","type":"article","venue":"Molecular Genetics and Metabolism Reports","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":12,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Eunice Kennedy Shriver National Institute of Child Health and Human Development; National Center for Advancing Translational Sciences; National Cancer Institute; National Institutes of Health; Mallinckrodt Pharmaceuticals; University of Washington; University of Pennsylvania; Alvin J. Siteman Cancer Center; Sucampo Pharmaceuticals; Institute of Clinical and Translational Sciences; National Niemann-Pick Disease Foundation","keywords":"Medicine; Adverse effect; NPC1; Lysosomal storage disease; Cholesterol; Niemann–Pick disease; Liver disease; Niemann–Pick disease, type C; Gastroenterology; Disease; Internal medicine; Drug; Pharmacology","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001012497,0.0002453177,0.00121454,0.0001321934,0.00004947276,0.00006679526,0.000144514,0.0001395837,0.00002916628],"category_scores_gemma":[0.0008593913,0.0001993428,0.0001759017,0.0003151102,0.000147233,0.0000516645,0.0002889129,0.0002246364,5.642928e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0000187251,"about_ca_system_score_gemma":0.0009500506,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001783625,"about_ca_topic_score_gemma":0.00004974693,"domain_scores_codex":[0.9970801,0.0002374732,0.001048553,0.0007113168,0.0005303365,0.0003921441],"domain_scores_gemma":[0.9979664,0.00009119381,0.0003220679,0.0007383412,0.0004292843,0.0004526988],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"randomized_trial","study_design_scores_codex":[0.6253014,0.03102148,0.00692506,0.0006016459,0.003265204,0.09485152,0.000801503,0.00007640809,0.06506424,0.0002886544,0.0001751349,0.1716277],"study_design_scores_gemma":[0.938776,0.004453782,0.01412388,0.0002420507,0.00342849,0.0006751699,0.00004146181,0.002636296,0.02465023,0.001017729,0.009343962,0.0006110094],"study_design_candidate":"randomized_trial","study_design_consensus":"randomized_trial","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9828523,0.009798294,0.00239943,0.0001807324,0.000185164,0.004341714,0.00003338712,0.000009786831,0.0001991984],"genre_scores_gemma":[0.9877701,0.003060352,0.008037744,0.0002219547,0.00009242451,0.0003515258,0.0001112347,0.00005653906,0.0002981575],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.3134746,"threshold_uncertainty_score":0.8128965,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04375647309428815,"score_gpt":0.3977924042026588,"score_spread":0.3540359311083707,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}