{"id":"W4392664758","doi":"10.1055/s-0044-1779185","title":"Efficacy and Safety of Fidanacogene Elaparvovec in Adults with Moderately Severe or Severe Hemophilia B: Results from the Phase 3 BENEGENE-2 Gene Therapy Trial","year":2024,"lang":"en","type":"article","venue":"Hämostaseologie","topic":"Hemophilia Treatment and Research","field":"Medicine","cited_by":2,"is_retracted":false,"has_abstract":true,"ca_institutions":"McMaster University; Thrombosis and Atherosclerosis Research Institute","funders":"","keywords":"Medicine; Genetic enhancement; Internal medicine; Phase (matter); Pediatrics; Gene; Biology; Genetics; Chemistry","routes":{"ca_aff":true,"ca_fund":false,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0004795054,0.0003273382,0.0005609809,0.0001195057,0.0001069428,0.00003717012,0.0001868451,0.0002160216,0.0002167489],"category_scores_gemma":[0.0003177226,0.0001566675,0.0000905054,0.000525078,0.000284865,0.0000915897,0.00009138186,0.0003893974,0.00001876935],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00009652806,"about_ca_system_score_gemma":0.0005945179,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0004468645,"about_ca_topic_score_gemma":0.0002749262,"domain_scores_codex":[0.9976665,0.0001857087,0.0005650231,0.0006787484,0.0004533911,0.0004506134],"domain_scores_gemma":[0.9976849,0.001373811,0.00007684506,0.0005980959,0.00009946775,0.0001668899],"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":[0.8655268,0.001082016,0.0006963386,0.0000948247,0.0005345463,0.002157646,0.001536747,0.0001502357,0.002165893,0.00002755123,0.00193977,0.1240877],"study_design_scores_gemma":[0.8603628,0.01990121,0.01489876,0.002438341,0.0004396619,0.0004954378,0.001192582,0.004885232,0.08339649,0.0004426565,0.01059168,0.0009552152],"study_design_candidate":"randomized_trial","study_design_consensus":"randomized_trial","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9838031,0.006595999,0.000008456221,0.005198892,0.0001204457,0.002280701,0.00145034,0.00007878799,0.0004632925],"genre_scores_gemma":[0.9900438,0.0072091,0.0005246106,0.0001970421,0.000273573,0.00006171557,0.0008763649,0.00003954257,0.0007742899],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1231324,"threshold_uncertainty_score":0.6388714,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.06854319924111586,"score_gpt":0.3452039801936729,"score_spread":0.2766607809525571,"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."}}