{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001879784,0.002277588,0.001939005,0.0004642695,0.0005315566,0.0009196457,0.0009628577,0.001818521,0.004758564],"category_scores_gemma":[0.001713554,0.0006965067,0.0007530334,0.0003436024,0.001416344,0.001013457,0.0003672942,0.002523836,0.0007887085],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006938947,"about_ca_system_score_gemma":0.001549651,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000515472,"about_ca_topic_score_gemma":0.00106741,"domain_scores_codex":[0.9993374,0.000366874,0.00003066173,0.0001045974,0.00004788621,0.0001126102],"domain_scores_gemma":[0.9990886,0.000238859,0.0001841391,0.00009742168,0.00006755299,0.0003233835],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.9137385,0.05718105,0.001213085,0.000395163,0.0003096894,0.0001547913,0.0001359467,0.0005628048,0.008058801,0.0001962857,0.001067542,0.01698633],"study_design_scores_gemma":[0.753282,0.2432031,0.001371438,0.00001640976,0.00006394574,0.00006126714,0.00002392905,0.0003601552,0.0009946502,0.0001082954,0.0005048421,0.000009997341],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9874402,0.0007896217,0.0007423676,0.0003997731,0.0002043034,0.00855984,0.0007054786,0.00009873882,0.001059476],"genre_scores_gemma":[0.9723806,0.001187099,0.002849566,0.001120331,0.0003185,0.01730264,0.001365056,0.00003664792,0.003439511],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.004758564,"threshold_uncertainty_score":0.01591897,"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."}}