{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001398309,0.000492771,0.0008272747,0.0001516926,0.0002260459,0.0005595704,0.0002975094,0.0007085415,0.001979162],"category_scores_gemma":[0.0009348125,0.0001367509,0.001047588,0.0001669332,0.0004294567,0.0004216581,0.0002050755,0.001033907,0.0002742044],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003351718,"about_ca_system_score_gemma":0.0004388564,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003990436,"about_ca_topic_score_gemma":0.0009537212,"domain_scores_codex":[0.99949,0.0002874705,0.00003394712,0.00006596013,0.00005058622,0.00007196604],"domain_scores_gemma":[0.999443,0.0001621165,0.0001398743,0.00002935609,0.00003462359,0.000190946],"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":[0.7312213,0.01092506,0.05347333,0.001536601,0.003893599,0.000303558,0.0003743486,0.0008237005,0.01782563,0.00060022,0.004329046,0.1746937],"study_design_scores_gemma":[0.368563,0.4349501,0.1768276,0.0002994346,0.003341732,0.0009243177,0.0002194539,0.0008783655,0.003658632,0.000614567,0.009655769,0.00006702264],"study_design_candidate":"randomized_trial","study_design_consensus":"randomized_trial","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9883206,0.008636565,0.0002376353,0.0004642828,0.00006375637,0.0003188074,0.000568935,0.00001978581,0.001369652],"genre_scores_gemma":[0.9948309,0.001987627,0.0004385324,0.000679999,0.0001271103,0.0004016242,0.0007990926,0.000005017351,0.0007300336],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001979162,"threshold_uncertainty_score":0.007395029,"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."}}